Issue 19 - April 2017

Fabry Focus e-NewsletterApril 1, 2017

Greetings!

Greetings and Welcome to Fabry Disease Awareness Month!

We are pleased to distribute this newsletter on the first day ofFabry Disease Awareness Month and on International Fabry Women's Day.

With many ongoing discussions about how to interpret diagnoses of classic and non-classic forms of Fabry disease, about access to treatment, about ongoing and newclinical trials for potential new treatments, and about many other important topics,Fabry disease awareness is more important than ever.

During April, please see our frequent face book posts, tweets, and other awarenessinformation and share it widely. Everyone can help to spread awareness!

... and let us know what you are doing for Fabry Disease Awareness Month so we canshare it with others.

April is also our primary annual fund raising month to raise funds for our manyprograms and services. As a 501(c)(3) charitable organization, we rely solely oncharitable contributions to continue our work to serve the Fabry community.

This month's newsletter includes:

April 2017 Fabry Disease Awareness MonthCommunity Outreach and EngagementOur Annual Fundraising CampaignNFDF Vital StatisticsNFDF Board of Directors and Staff Strength and CourageAfter All These Years TogetherMy Health Handbook KitsNFDF Family Assistance Program (FAP) Trials and Tribulations This Year's NFDF Conference and Camp7th Annual Fabry Family Education Conference8th Annual Charles Kleinschmidt Fabry Family Camp2017 Volunteer ApplicationsFSIG Conference - April 28 -30, 2017

Other Upcoming NFDF MeetingsDesignation of Disease Causing MutationsImportant Studies - Please Participate We Mourn the Loss of our FriendsReconition of 2016 Conference and Camp VolunteersAn Essential Ingredient to our Success is You!Shining Star Campaign acknowledgementsUntil Next Time

We are supporting individuals with Fabry disease and their families in as many waysas we can and are expanding our programs and services as opportunities arise.

Sincerely,

Jerry Walter

April 2017 Fabry Disease Awareness Month

Beginning today through the end of Fabry DiseaseAwareness Month, please share our frequent posts andtweets. Our face book and twitter pages are www.facebook.com/FabryDisease andwww.twitter.com/FabryDisease1. This month is a greattime for everyone to catch up on information you mayhave missed and to take an opportunity to shareinformation with others. Start by sharing our FabryDisease Awareness Month flyer to the left. By sharinginformation, you may help someone find answers or getthe help they need.

Also, by increasing education and awareness we can allhelp to improve disease recognition, diagnoses,understanding, and proper management to give peoplewith our disease an opportunity to live significantlybetter and longer lives!

We apologize for the disruption this year in our annualgovernors' proclamation program! Normally, from January through April we coordinate withcommunity representatives in each state to request an official governor's proclamation forApril as Fabry Disease Awareness Month. Due to having more projects than we have staffand time, we were unable to perform our community coordination this year. Next year, we'llstart early and resume this important program. Thanks for your understanding!

Community Outreach and Engagement

The NFDF is engaged in many activities

In addition to the daily support we provide to our community,we are always engaged in many other activities.

Here are some examples of our recent activities since ourlast newsletter was distributed.

August 2016: Jerry spoke at an FDA Panel Meeting advocating for approval ofthe SEEKER newborn screening system which has since been approved.

August 2016: We hosted Fabry family meeting in Salt Lake City. August 2016: Jerry participated in an FDA meeting with Jack from FSIG andothers (Dr. Rob Hopkin, Dawn Laney, and others) about Fabry clinical trials.

September 2016: We hosted the NFDF annual conference and camp.

October 2016: Took time to catch our breath after the conference and camp.

November 2016: We hosted Fabry family meetings in Las Vegas andPhiladelphia.

December 2016: We held a semi-annual NFDF Board of Directors meeting.

January 2017: Jerry spoke at an Amicus Therapeutics employee Lunch andLearn in Cranbury, NJ.

February 2017: Jerry attended 2 days of the Lysosomal Storage DiseaseNetwork (LSDN) World Symposium in San Diego.

March 2017: Jerry spoke at a Protalix Biotherapeutics Clinical Trial Investigatorsmeeting in NYC.

Other recent significant activities include:

We responded to many requests for assistance from our Urgent and UnmetNeeds (financial assistance) program .

We developed and began mailing our "My Health Handbook" kits aroundthe world. We have distributed about 500 and are making 500 more.

We rebuilt our Find A Doctor search tool on our website and are addingphysicians treating Fabry disease. If you have Fabry disease and yourprimary Fabry-care physician is not listed, please let us know.

We placed updated verions of our NFDF Program Handout and FabryCommunity Landscape documents on our website.

We developed a Document Archives section to our website where we areplacing past newsletters and other notable documents.

We added new calendar items including our conference and camp dateswith details.

We facilitated completion of several surveys to increase the understandingof Fabry disease and to aid in clinical trial development and participation.

We developed a new Fabry disease awareness video we will release thismonth.

... and much more!

Our Annual Fundraising Campaign

Along with Fabry Disease Awareness Month comes the beginning ofour primary annual fundraising campaign. The amount of charitablecontributions we receive each year determines the number ofprograms and services we can provide to our community.

You, our donors, make our programs possible!

We can't do it without your support.

We are very fortunate to have a matching donationcampaign this year to help us reach our April fundraising goal of

$25,000.

Barbara Fitzpatrick has again this year offered to match the first $3,000 of yourdonations toward our goal.

Thank you Barbara for your continued very generous support!

This year, we would especially like to raise more funds to providesupport to financially challenged individuals who have unmet medicalneeds (not Fabry treatment itself), unmet basic living expenses, andunmet transportation needs.

The number of requests we receive to support urgent and unmet needs is increasingdratmaticlly over past years as more and more families are diagnosed with Fabrydisease.

You may donate via the Donate Now button above or by sending a check to NFDF NCOffice, P.O. Box 1325, Carrboro, NC 27510.

We appreciate your generous contributions!

NFDF Vital Statistics

Using the latest communicationstechnologies, we have tremendousreach in the Fabry community:

Here are some of our vitalstatistics:

Face book Likes (www.facebook.com/FabryDisease) = over 2,300 people

On Facebook: Connected to people with Fabry disease in 47 countries

Greatest reach for a single post (a few years ago) = 27,000 people in 3 days

Second best face book post (February 28, 2017) = over 19,000 people in a day

Connected to adults with Fabry by email: approximately 1,000 and growing

NFDF Fabry Symptoms Video: www.YouTube.com/TheNFDF = 14,600 views

Other NFDF videos: 631, 695, 1985, 2368, 9463 views as of 3-31-2017

Highest NFDF Patient Reported Outcomes Survey (PROs) responses for asingle survey = over 400 adults with Fabry (learning from the Pros)

Fabry Focus e-Newsletter subscriptions: 1277 subscriptions although we knowsome are duplicates we are cleaning up. Subscribe at this link: Subscribe

Website regisrations = about 400 (restarted in 2016 to track My HealthHandbook kit distribution) Register at this link: Register

My Health Handbok Kits distributed = almost 500 sent ... making 500 more

2016 annual conference attendance = 250 attendees (including 118 adults andkids with Fabry and 93 family members)

2016 Camp and conference volunteers = 47 volunteers from the Fabrycommunity plus over 40 more from Victory Junction for camp

We are just getting started!

NFDF Board of Directors and Staff

We have not formally introduced you to Susan.

Although some of you may recognize Susan Spinks (on theright next to my wife Angela) from checking in at ourannual conference for the last couple of years, we havenot made a formal introduction.

We are very happy to have Susan's help as a part-timestaff member performing many critical tasks such asconference administration, NFDF membership/constituency management, financial managementsupport, and My Health Handbook Kit and awareness

materials distribution, to name a few things Susan does so well.

Thank you to Susan as she helps tremendously to make us more efficient and timelyas we serve the Fabry community.

We would also like to thank the newest members of our Board of Directors fortheir willingness and commitment to serve, and for the expertise and experience they

bring to improve the management and oversight of our organization.

Welcome and thank you to Ms. Tracy Myatt, Ms. Melanie Wilson, and Mr. StevenRothman!

... and of course, thank you again to Mr. Derek Halberg, Dr. Douglas Warnock, Ms.Elizabeth Gaffin, and Dr. Tracey Krasnow who have all been with us since thebeginning, for your years of service on our Board of Directors.

We are so fortunate to have a tremendous strategic and policy leadership team and adedicated and motivated staff!

Please see more about our board and staff in the About the NFDF tab at the top of ourwebsite homepage.

Strength and Courage

The courageous acts of others in our community gives us great hope!

Brandon gets a kidney from his older brotherPhil. See the whole story at this link.

http://www.fox2detroit.com/news/local-news/221220007-story

Darren makes history in a gene therapy trial inCanada. Read the story at Darren's story. Our friend Darren is a past president of theCanadian Fabry Association and a past volunteer atour annual camp. His courage in beginning this trialfor the benefit of all people with Fabry disease isincredible. Thank you, Darren!

Last month, Cheryl courageously underwent a hearttransplant. Cheryl has been struggling with heartproblems for many years. The progressive decline inher health made simple things very difficult andchanged her life tremendously. But, despite thehardships, Cheryl has always remained positive andoptimistic, and is always willing to help others. She is

a compassionate voice and a sound advisor in our social networking community.Cheryl, thank you for all you do and for the great example you provide. Best wishes toyou and Bob in your exciting after-transplant life!

After all these years together ...

Thank you and congratulations to KathleenCoolidge!

Kathleen is retiring from her position as the Directorof U.S Patient Advocacy for Sanofi - Genzyme.

The photo above shows Kathleen (front row - 2ndfrom left) having fun as a volunteer family crew chief at Victory Junction. Kathleen'svolunteer efforts at our annual conferences and camps are just small examples of hermany contributions to the Fabry community.

We have worked with Kathleen for more than 10 years. We really appreciate hertremendous dedication to and steadfast support of people with Fabry disease and ourfamilies.

Farewell and good luck on your next journey, Kathleen. We will miss you! We havebeen through a lot together.

Jerry Walter, on behalf of the NFDF and the Fabry community

My Health Handbook Kits If you have Fabry disease, do you have your kit?

Are you prepared for medical appointments andemergencies?

The kit includes:

a durable, stylish carrying case our new My Health Handbook a 8 GB credit card style USB drive alert card wristbands and other awareness materials

For the U.S and international Fabrycommunity

Contact Jerry at jerry.walter@fabrydisease.org for information about how toobtain kits.

If you work at a Fabry clinic, please request a sample kit to show yourpatients and a flyer to distribute to your local patient community.

Don't forgot about our Family Assistance Program (FAP)

Family Assistance ProgramThe NFDF is proud to continue our free, confidential,24/7 Family Assistance Program for individuals in theU.S. with Fabry disease and their family members.

The NFDF contracts the confidential professional counseling services ofKathleen Greer Associates (KGA) who are standing by ready to take yourcalls. Any personal issue is appropriate. The service is completely confidential.The NFDF only receives summary data about the assistance provided by KGA,no personal information.

To reach a counselor 24/7, call 800-648-9557. Participants can also access the member section of the KGA website through theusername NFDF and the password 9557 (the last 4 numbers of KGA's 800number, 800-648-9557).

See more details in the Featured Programs section on the NFDF websitehomepage at www.fabrydisease.org.

This program is funded in part by Sanofi Genzyme.

Trials and Tribulations

Clinical trials for investigational Fabrydisease treatments increase.

Understandably, it is difficult to obtain informationabout investigational treatments in the clinical trialprocess until the results of the clinical trials arepublished and the investigational medications are

approved. Meanwhile, we are happy to report there are several clinical trial and pre-clinical trial activities in progress.

By now everyone should know that the new Amicus oral chaperone therapy for Fabrydisease, Galafold (TM)/Migalastat, was approved in countries outside the U.S. but notyet in the U.S. The FDA required Amicus to undergo additional clinical trial work.

Despite the hopefully temporary setback for Amicus in the U.S., we are optimistic thatwe have a future that includes several Fabry disease treatment options available inthe U.S. and abroad.

Here is a short list the possibilities we know about, but probably not all inclusive:

Amicus - oral chaperone therapyProtalix - enzyme replacement therapySanofi Genzyme - substrate reduction therapyActelion - substrate reduction therapySangamo - gene editingAvobio - gene therapy

University of Calgary and the Alberta Children's Hospital - gene therapy

We met with the first five companies listed at the recent World Symposium in SanDiego. As clinical trials proceed and information becomes available we will pass iton.

You can learn about the ongoing Protalix trials at http://www.fabrynext.com

Actelion provided a survey for adults with Fabry to gain a better understanding ofpatient perspectives. The survey is available at this link.

Our future looks much brighter!

This year's NFDF conference and camp

Save the Dates! - See Details Below

The NFDF annual conference and camp have provided wonderful opportunities for

families to get to know each other, to learn from physicians and from each other, to

play together, and to develop mutual support relationships that last a life time.

Thank you to everyone who helped to make our conferences and camps possible!Please see the acknowledgements to our major sponsors, individual donors, andvolunteers later in this newsletter.

7th Annual Fabry Family Education Conference

Thursday, 28 and 29 September, 2017

Individuals with Fabry disease and their family members are invited to attend theannual Fabry family education conference in Greensboro, NC. Lodging andmeals are provided. Travel assistance is provided based on available resources.

In 2016, 250 people attended the conference including individuals with Fabrydisease (118 adults and kids), family members (93), physicians and clinic staff,conference and camp volunteers, speakers, and industry representatives.

We fly attendees to Greensboro, NC as early as possible on Thursday to enablemaximum participation in informal Thursday afternoon and evening activities. OnFriday, we have a full day of physicians and other speakers providing educationalpresentations. Late Friday afternoon, we transport families going to camp tonearby Victory Junction. Attendees not going to camp after the conference departfor home on Friday evening or Saturday morning depending on trip durations andavailable flights.

To do list:

Contact Jerry Walter at jerry.walter@fabrydisease.org as soon as possibleto let us know you wish to attend the conference. We'll contact you to obtainyour travel information and to provide conference details closer to the datesof the conference.

_____________________________________________________________

8th Annual Charles Kleinschmidt Fabry Family Camp

Friday, September 29 to Sunday, October 1, 2017

Families with at least one child age 6 to16 with Fabry disease are eligible for theentire immediate family to attend camp. Airfare, ground transportation, lodging,and meals are provided.

Since 2010, we have hosted 115 families at Victory Junction camp. Becausefamilies are able to attend camp for more than one year, we have provided a totalof 284 family camp experiences to date. For information about camp and for application instructions please visitour website's "Upcoming Events" (calendar) section. Please read theinformation in the July 1st calendar entry titled "Families andvolunteers submit camp applications", and also read the follow-onConference and Camp calendar items on their respective dates. Please visitwww.victoryjunction.org to learn about this amazing place we have adopted asour second home.

To do list:

Contact Jerry Walter at jerry.walter@fabrydisease.org to let us know you

wish to attend. We'll contact you for family attendee information.Make doctors appointments for your kids now to have the medical formssigned (for kids with Fabry), and to obtain immunization documentation (forALL kids under 18) so you have them before July 1st. Submit your online camp application on July 1st.In September, get to the airport on time and we'll have some fun together!

Soon, we'll send more information to everyone who has contacted us.

Important note:Because we need to get attendees approved for camp by Victory Junction beforewe can purchase plane tickets, we will only accept online applications for campuntil July 31st, one month after the application process begins.

To see photos of our 2016 camp visit our face book page at www.facebook.com/FabryDisease in the "2016 Charles Kleinschmidt FabryFamily Camp at Victory Junction" photo album. You can enjoy more than 600photos of last year's camp. For more information, contact Jerry water at jerry.walter@fabrydisease.org or800-651-9131.

2017 Volunteer Applications

2017 Volunteer Application Process

We need you! Please join us for this fun, gratifyng experience for the benefitof families with Fabry disease attending camp. The Victory Junction (VJ)volunteer application process also begins July 1, 2017. The somewhatrigorous volunteer approval process has several steps so please start early.Volunteers must be at least 19 years old.

Volunteers may attend a portion of the conference on Thursday and Fridaymorning and have lunch with us on Friday before going to camp ahead of ourfamilies or they may go straight to Victory Junction to arrive by 2:00 pm on Fridayafternoon. We'll clarify the offical arrival time later. Departure from camp isSunday at noon.

Like the camp attendees, volunteers must also have a VJ medical form signed byyour physician and must provide immunizaion documentation. It can be a littleharder for adults to locate immunization records, so titer tests may be required toshow adequate levels of protection.

You can find the Volunteer Physical Exam Form and the Immunization Policy inthe July 1st "Upcoming Events" (calender) entry on our website.

In addition to camp volunteers, we also need a few volunteers to support the

Thursday/Friday conference. Please contact Jerry if you are able to support theconference along with or instead of the camp.

To do list:

Please contact Jerry at www.fabrydisease.org to let us know you would like tovolunteer. Soon, we'll provide additional information to everyone who hascontaced us.

Thank you all so much! We look forward to seeing you in September!

See the list of 2016 Conference and Camp Volunteers later in the newsletter.

Jerry Walter

FSIG Conference - April 28 - 30, 2017

Registration for the FSIG Expert FabryConference is EXTENDED!

If you have not registered yet please do so before

Noon on April 4, 2017!

Send FSIG your Best Medical Professional and Best Caregiver Application AwardNOW.

A few rooms are still available at Embassy Suites Cincinnati - RiverCenter 10 East Rivercenter Blvd Covington, KY 41011

Friday and Saturday nights lodging are paid for by FSIG. If you have not alreadyregistered all you need to do is complete the registration and take care of your travel.Register at http://www.cvent.com/d/jvq50h, or call 866-30-FABRY, or emailsupport@fabry.org The weekend includes - Friday night the 28th Dawn Laney from Emory will have her Fabry 101 class at 5 PM We will have great food at our Fabry Social starting at 6 PM

Saturday the 29th We have a full day planned including meals and information starting at 8:30 AM

We will take a break from 5:00 PM to 6:30PM

Dinner and a motivational speaker starting at 6:30 PM

Sunday the 30th After breakfast Sunday morning a final interactive session from 9:00 to 11:00 AM will

cover Fabry related Psychological issues.

Come join the fun in meeting new and old friends.

Other Upcoming NFDF Meetings

We hope to see you in yourneighborhoods!

The following NFDF meetings are scheduled:

May 6: Fairfield, California Fabry Family Meeting (Jelly Belly Company)

May 7: San Jose, California Fabry Family Meeting

September 28-29: Greensboro, NC Annual Fabry Family Education Conference

September 29 - October 1: Randleman, NC (Victory Junction) Annual CharlesKleinschmidt Fabry Family Camp

Date/Location to be determined: Virginia/Maryland Fanry Family Meeting

Additional 2017 meetings to be determined at a later date

Designation of Disease Causing Mutations

Classic and Non-Classic Fabry Disease

Disease Causing Mutations and Non-DiseaseCausing Mutations

Caution would be prudent!

There seems to be an ongoing debate about whethersome non-classic family mutations such as the A143Tmutation are a cardiac variant, a non-disease causingmutation, or something else.

Some researchers seem to want to rush to the conclusion that the A143T mutation isnon-disease causing. Further investigation is probably warranted to avoid adverseconsequences to families who have the A143T mutation and documeted GL-3 lipidaccumulation. At least one case has been discovered and there may be more.

In research using small representative populations, the critical thinking

model can sometimes go astray. Assumptions can become facts, onlylater become myths.

After all, it took a very long time and many mistakes before researchers realized Fabrydisease is not an X-linked recessive disease and symptomatic females are theexception, not the rule.

If your family has been diagnosed with the A143T mutation, please let us know atjerry.walter@fabrydisease.org so we can connect you with any research to clarify thecharacteristics of your mutation. Your personal information will not be shared.

Important Studies - Please participate!We have several study opportunies to share with you.

Dr. Bill Wilcox, Dawn Laney, CGC, andEmory Genetics team do so much for us!

We are happy for the opportunity to give back to Emory and to ourcommunity. These studies benefit us all.

Please participate in the studies below that are right for you.

Study 1: Help us learn about how kids with Fabry grow!

The Emory University Department of Human Genetics in Atlanta, GA is currentlyenrolling interested MEN living with Fabry disease between the ages of 16 and 40years in a study called, "The Impact of Fabry disease on Growth in Males" or IFOG.This study involves a medical record review and one questionnaire investigating theimpact that Fabry disease, enzyme replacement therapy, and gastrointestinalsymptoms have on growth in males with Fabry disease. The study can be done atregular doctor visits and the questionnaire can be completed electronically, on thephone verbally, or on paper via email/regular mail. You do not have to travel to Emoryto participate in the study. No stipend will be provided for participating in this study.

If you are interested in participating or would like more information please contactKait Miller via phone (404-778-1014) or email (kaitlyn.marie.miller@emory.edu) orDawn Laney via phone (404-778-8518) or email (dawn.laney@emory.edu)___________________________________________________________

Study 2: Are you living with, a family member of, or caring for someone livingwith Fabry? If so, please consider being part of our study! It includes completing an 11 questionresearch survey, visiting an educational website, and the option of entering a raffle towin one of three $250 gift cards! (You do not have to participate in the study to enterthe raffle.)

If you'd like to learn more about this study or to participate, visit this URL:https://tinyurl.com/thinkgenetic

If you have questions contact Morgan Simmons atmorgan.frances.simmons@emory.edu or Dawn @ dawn.laney@emory.edu____________________________________________________________

Study 3: The MOPPet Study Do you have a little boy diagnosed with Fabry? Please help us understand how Fabryaffects babies and toddlers

The Emory University Department of Human Genetics in Atlanta, GA is currentlyenrolling interested babies diagnosed with Fabry disease before 3 years of age andtheir qualifying family members in study called "A Prospective, Multicenter Pilot StudyOf Fabry Disease Clinical and Biochemical Findings in Young Pediatric Patients Study"or MOPPet. This study involves urine collection, physical exam, and twoquestionnaires investigating the impact of living with Fabry disease as babies andyoung children over the course of four years. The study can be done at regular doctorvisits and the questionnaire can be completed electronically, on the phone verbally,or on paper via email/ regular mail. You do not have to travel to Emory to participatein the study. A $30 stipend is provided for each enrolled individual at each visit.If you are interested in participating or would like more information please contactDawn Laney via phone (404-778-8518) or email (dawn.laney@emory.edu).

_____________________________________________________________

Do you have any gastrointestinalsymptoms such as abdominal pain,diarrhea or nausea?

If yes, you may be eligible to participate in a research study on people with Fabrydisease.

Who? Adults over 18 years old with Fabry disease who havegastrointestinal symptoms

Why? To learn more about the gastrointestinal symptoms inFabry disease

How? To learn more about this study, contact the studycoordinator at Massachusetts General Hospital atrzsolomon@partners.org or at 617-724-0480.

This study is voluntary. Participants will be compensated for their time. The study willuse questionnaires to learn more about your symptoms and a device to study themovement of contents through your gut.____________________________________________________________________

We were contacted by a company, Perkin Elmer, who aredeveloping a newborn screening test for Fabry. They are inneed of neonatal dried blood samples for a clinical studyused in support of an FDA submission. FDA clearance isneeded to market the screening test to newborn screening

laboratories in the US and around the world.

The company has asked for our help to identify any children born in California whohave been tested positive for Fabry in the last fifteen years. If you would be willingto share your leftover child's newborn blood spots kept in the state's biobank for thisproject, please contact Jerry at Jerry.walter@fabrydisease.org for ddditionalinformation.

Personal information regarding your child will remain anonymous and will not beshared with the company.

Every little thing we do to promote early diagnosis of Fabry will allow for prompt,more effective treatment and will enable the affected children and their families tohave a better quality of life.

Thank you!

We Mourn the Loss of our Friends

We are deeply saddened by the loss ofseveral members of our community in recentmonths. Please keep their families in yourthoughts and prayers. Farewell friends ... Restin Peace.

Notes by Jerry Walter: The more connected our community becomes, the morepersonal our losses become.

Lauren Zeitels, MD, PhD, 32, Boston, Massachusetts About Dr. Lauren Zeitels

We recently worked with Dr. Zeitels by facilitating completion of a survey for aFabry disease study she was working on at Massachusetts General. We werevery excited to have such an accomplished physician interested in Fabry diseaseand supporting the Fabry disease community. While our paths only crossedbriefly, we realize we lost a very special person and physician with her suddenpassing. We are deeply saddened by this tragic loss.

Dave Costa, 56, Knoxville, TennesseeAbout Dave

Dave and his wife Linnae were annual camp volunteers. I have always felt a

strong connection to Dave. He was a kind, thoughtful, and caring man who it wasa pleasure to know.

Andrew Harris, 53, Sheffield, EnglandAbout Andrew

Andrew and I began emailing each other in 2013. We oftenexchanged stories about how various Fabry symptoms were affecting us. He wasa strong, courageous, upstanding guy who I really looked forward to meeting inperson when we had the chance.

Michael Sico, 59, Southwick, MassachusettsAbout Michael

I didn't meet Michael in person, but I corresponded with some ofhis family members. Based on the outpouring of tributes to Michael, he was lovedand respected by so many people and will be missed tremendously.

2016 Conference and Camp Volunteers

Thank you to our amazing volunteers!

In 2016, the Fabry community provided 36 of the 60 volunteers needed for camp.Victory Junction provided the rest. The photo above shows Team Genzyme whoassisted with 12 volunteers. Our Fabry community volunteers are individuals withFabry disease healthy enough to support camp, friends, family members, clinicstaff, and industry staff. Our 2016 camp volunteers:

Abbe GoldingAlexis Shepherd Amanda Hodgkins Amber BrittonAndy KleinschmidtAngela WalterBrian AltonCasey Geddes Cheryl BarcombDan Kuusisto

Dave CostaDave JacobDawn Laney Diana Moore Edie Dodson Eric RiceHolly MayJulia AltonKasia MackoKatie Reese Kelly GroveKevin FowlerLaura KlineLinnae CostaLoree Bren Marleni Arvelo-SaillantMegan Olsen Morgan Simmons Nancy NgothoPage Bales Paul RakoskiDr. Rob HopkinSteve RothmanTim FalencikVictoria DukeXenia Gramata

The following people, in additon to many of the camp volunteers above,supported various aspects of the conference to provide our families with a verysuccessful meeting and a great experience.

Connie WehmeyerKatie LongLance WebbLanna MoxleyLisa BerryManish MaskiMario AguiarMelanie WilsonNita PatelSarah NelsonShane Palmer Susan SpinksTracy Myatt

Thank you so much to all of you! The contributions of your time, your hard work,your kindness, and your remarkable spirits inspire us all!

Jerry Walter

An Essential Ingredient to our Success is You!

Shining Star Campaign

Be the brightest star you can be!

Thank you to our generous shining stars for this period!(includes donations received after our June 2016 newsletter was

published.)

Our Sirius Sustainers (Major Contributors) (Read about our *Sirius Sustainer Program below)

The Kleinschmidt Family FoundationSanofi - Genzyme Corporation

Shire Human Genetic TherapiesAmicus Therapeutics

Protalix Biotherapeutics

Champion Level Donors ($1,000 or more)

Ben BeydaJeffrey D. MinelliBernadette Beyda in honor of BenMichelle Nunns- JensenBarbara WhitenerMichelle JensenRon Dukenski to support the Fabry Victory Junction CampTodd Parsons

Sustainers: (Automatic Recurring Donations)

Jaime Hanrahan in honor of the Bohn Family

Leader Level Donors ($500 to $999)

Stacy Ho (donation matched by her company's matching gift)Kenneth Diblin in honor of Patricia ArtznerVivian Pace-Smith in honor of Jerry and Angela WalterCatherine Abrahams in memory of Mark Buske

Dr. Robert DesnickRon Dukenski to support the Fabry Victory CampDavid Beyda

Advocate Level Donors ($100 to $499)

Alan and Tracy Schwartz in memory of Joey WallaceThomas Wallace in memory of Joey WallaceGary and Sandy Mesich in memory of Joey WallaceMartin and Lisa Levine in memory of Joey WallaceFrederick and Rita Burke in memory of Joey Wallace Richard and Margaret Sullivan in memory of Joey WallaceMark and Carol Chaikin in memory of Joey WallacePaula and Samuel Agger in memory of Joey WallaceAllan Dosik in memory of Joey WallaceCraig Bernstein in memory of Joey WallaceStephen Mitchell in memory of Joey WallacePaul Sisco in memory of Joey WallacePaul Ahearn in memory of Joey WallaceOrenberg Law Firm in memory of Joey WallaceAlan M. White in memory of Joey WallaceDon and Randy Greenberg in memory of Joey WallaceJohn and Bonnie Peterson in memory of Joey WallaceGary Melnick in memory of Joey WallaceJoyce Voelker in memory of Joey WallaceSarah Knorr in memory of Joey WallaceSheldon Dosik in memory of Joey WallaceDavid Dison in memory of Joey WallaceSteve Smith in memory of Joey WallaceCynthia Hodges in memory of Joey Wallace

Fred and Gerardo Sico in memory of Michael SicoMargaret Kelliher in memory of Michael SicoJoanne Sico in memory of Michael SicoJohn Moody in memory of Michael SicoRoots Gymnastic Center in memory of Michael SicoWestfield Public Schools Custodians Assoc. in memory of Michael SicoDouglas and Dayna Puza in memory of Michael Sico

Andrew Marshall Tamara Klein Shulman David Backin the name of The Back Family - I have donated a total of $500 to the NationalFabry Disease Foundation.Steven Rothman in honor of all the good work of Angela and Jerry WalterSusan and Jim Randin honor of Bernadette Beyda's birthdayJonathan Katz in memory of Betty HillTina Stetz Barbara Sharpin honor of Joe DuganDavid Back in honor of the Back FamilySteven Rothman in honor of Bruce & Sue's 60th BirthdaySt. Patrick Church in memory of Mark Buske Andrea Parker -thank you to all of my family and friends who put this donation together!Steven Rothman in Honor of Jerry and Angela Walter for all the good work you do.Beverly London in memory of Joe Edwin DuganSherry Tranchemontagne in memory of Richard BealandJeanne Becker in honor of Jim and Judi LilliganDeborah Schultz

Donna St. Denny Manvil and Nan Hendrickson in memory of David Costa Emory University School of Medicine in memory of David CostaJerry and Angela Walter in memory of Dave Costa Kurt Langenwalter in honor of David Costa and familyEdie Dodson Shawn Satterfield in memory of Brian LeanderPaula Hart in memory of Mark S. BuskeSheldon Dosik John Fox in honor of Marky Lo FaroBernadette Beyda in honor of our very dear friend Susan on the occasion of her birthday Eve Rose Rubell iIn memory of Josh RubellEllen and Mike Shalerin memory of Josh RubellDennis and Kathy Duffy in memory of Scott PagnottoSathya Amouretti Eileen Walsh Green in memory of Robert Polo Hope Snyder in honor of Josh RubellKimberly Kondak in honor of John & Anne NorsworthyCatherine Sargent in honor of Chad's Father, Tom Smith Jeanne Becker on behalf of Bob and Gayle BeckerKyle ButlerSteven Rothman in honor of Jerry & Angela WalterSherry Tranchemontagne in memory of Richard BealandEmory University School of Medicine in memory of Wayne KennedyDavid and Marcy Goldman Tresten Lada Brian Ahlquist in honor of Scott Tomlin Pagnotto

Supporter Level Donors (up to $99)

Joseph and Karen Wzorek in memory of Michael SicoTimothy Pemberton in memory of Michael SicoSamuel and Lauri Sico in memory of Michael SicoJames and Carol Demeo in memory of Michael SicoHighland School Sunshine Fund in memory of Michael SicoElaine Kuzmeskus in memory of Michael SicoMichael and Diane Camerota in memory of Michael SicoJoseph and Sheila Long in memory of Michael SicoTom and Carolyn Blaisdell in memory of Michael SicoJoanne and Jerome Scully in memory of Michael SicoMaureen Wrobleski in memory of Michael SicoDaniel and Liane Howard in memory of Michael SicoStephen Oleksak in memory of Michael SicoJames and Penelope Vincent in memory of Michael SicoDianne Magdycz in memory of Michael SicoNeva LeSage in memory of Michael SicoJudith and Timothy Tatro in memory of Michael SicoEleanora and Samuel Sico in memory of Michael SicoWestfield Education Association in memory of Michael SicoDaniel Tenero in memory of Michael SicoRobin Sheldon in memory of Michael SicoBrian and Katherine Wailgum in memory of Michael SicoAntonia and John Theroux in memory of Michael SicoCheryl Rothermel in memory of Michael SicoPaul and Joanne Romani in memory of Michael SicoSusan and Michael Regensburger in memory of Michael SicoMary Cullen in memory of Michael Sico

Kevin and Jacqueline McGettigan in memory of Michael SicoTimothy Gove in memory of Michael SicoJudith Maunsell in memory of Michael SicoTim and Gloria Howard in memory of Michael SicoKathryn Chandler in memory of Michael SicoJohn and Mary Morrissey in memory of Michael SicoBrian and Cheryl Richards in memory of Michael SicoCynthia Bullard in memory of Michael SicoThomas and Rosemary Gavin in memory of Michael SicoStanley and Marion Zomek in memory of Michael SicoKaren Crafa in memory of Michael SicoSandra Wantuch in memory of Michael SicoStefan Czaporowski in memory of Michael SicoMark and Jeanne Morin in memory of Michael SicoSheila and Michael Zagula in memory of Michael SicoDiane McKnight in memory of Michael SicoPatricia Galanski in memory of Michael SicoPatricia Marotte in memory of Michael SicoBonnie Sico-Russell in memory of Michael SicoAndrea and Benjamin Bryla in memory of Michael SicoLaurence and Elizabeth Hunt in memory of Michael Sico

Cynthia Hodges in memoty of Joey WallaceMary Alice Raeder in memoty of Joey WallaceKenneth Ende in memoty of Joey WallaceFrances Pennell in memoty of Joey WallaceKaren Flippo in memoty of Joey WallaceAmy Goldman in memoty of Joey WallaceEmily Cox in memoty of Joey Wallace

Christina Van Fossan in memory of Mark BuskeStaci Riera in memory of Mark BuskeClinton Dental Associates in memory of Mark BuskeJudith Gulley in memory of Mark BuskeAlicia Arendt in memory of Mark Buske

Loretta Turnipseed in memory of Mark PoloJoan Allison in memory of Mark PoloDiane Gurr in memory of Mark PoloPatricia Portanova in honor of the Polo family

Eve Rose Rubell in memory of Reina RubellAlla and Alex Berman in memory of Josh RubellJacqueline Ta in memory of Josh RubellJoy Blevins in memory of Josh RubellRuth Shulman in memory of Josh RubellWendy Gary in memory of Josh RubellAndrew Todd Berman in memory of Josh RubellDonna Malamud in memory of Josh RubellHoward Serbin in memory of Josh RubellMeyer and Harriet Press in memory of Josh RubellEve Rubell in memory of Josh Rubell

Dianne Short in honor of Lenny GoberArlene Yablin in honor of Lenny Gober

Lisa Foley in honor of Tatiana BergstromSarah Waite in honor of Tatiana Bergstrom

David Surgan in honor of Ben BeydaJordan & Jessica Lazarus in honor of Ben BeydaHarriet Cheney in honor of Bernadette Bayda's birthday

Beth Kaplan in honor of Billy's Uncle RayPatty Caulden in honor of Billy's Uncle RayBrett Sutherland in Honor of Billy's Uncle Ray

Cheryl Barcomb in memory of David CostaTim and Katrina Rieger in loving memory of David Costa

Rickie Yeager in memory of Thomas E. SmithJames and Anne Willemsen in honor of Susan LongSuzanne GravelSteve RoyDavid JohnstonJustin GouldKen SeligmanLinda StrangeJean RoederChristopher SmithJoseph and Beverly FranzDora CorelliJason WeingartenSamir Balwani

*Sirius is the brightest star (in fact, star system) in the Earth's night sky. Our Sirius categorydonors make major contributions to provide our annual funding base on which to build. OurSirius sustainers provide the starting point to make our programs possible.

Thank you to all of our wonderful donors for your tremendous support! Every sizecontribution adds up to make a huge difference. You have our deepest gratitude forbeing the brightest stars you can be! Together were better!

Until Next Time

Thank you all for your continued participation and support! Pleasecontact me anytime at the phone number or email address below.

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Proudly serving the Fabry community!

Sincerely, Jerry Jerry Walter, MSColonel, U.S. Army (Retired)Founder and PresidentNational Fabry Disease Foundation

www.fabrydisease.org

Copyright © 2016 All Rights Reserved.

| 800-651-9131 | jerry.walter@fabrydisease.org | 4301 Connecticut Ave. N.W.

Suite 404Washington, DC 20008