NORD® Conference Registration FeesNORD Member $ 135.00NORD Non Member $ 170.00Additional attendee from same $ 50.00family or organization (over age of 12)

One-Day Visit $ 75.00Total Amount Enclosed: $ ______

Please make all checks payable to NORD

If you prefer to use a charge card, please fill in below:

� MasterCard � Visa � Amex � Discover

Card Number _____________________________________

Expiration Date ____________________________________

Name on Card (print) ________________________________

Signature __________________________________________

Dr./Mr./Mrs./Ms. _______________________________________________________________________________________________First Name Last Name

Title/Affiliation ________________________________________________________________________________________________(Only applicable if you will be representing a support group, organization or company)

Mailing Address _______________________________________________________________________________________________

City ___________________________________________________ State __________________ Zip Code ____________________

Daytime Telephone Number ______________________________ Email Address ______________________________________

If you need additional registration forms, please photocopy this form and fill one out for each person who will be attending.

Please indicate (if applicable):Have you attended a NORD Conference before?� Yes: Year or Place: __________________________________� No

What is your relationship to the person with the rare disorder or disability?� Self � Parent � Spouse � Sibling� Relative or Friend � Other: _____________________

� Yes, I have special needs (handicap-equipped room,hearing/visual aids, dietary restrictions, etc.). If so, please specify. (Note: If you are in need of a specially equipped room, please advise the HyattRegency Bethesda when making your reservations).________________________________________________

� Yes, please list my name for any Conference NetworkingPrograms available under the following disorder:____________________________________________________

� Yes, I give NORD permission to indicate the above disorder on my nametag and registration materials.

� Yes, I would like to be considered for a partial scholarship (registration fee) for the NORD Conference.My written request is enclosed.

Please indicate if you will be attending the following:Continental Breakfasts � Friday � Saturday � SundayLuncheons � Friday � Saturday

Send to: NORD • Attn. Donna Bolton-Jackson P.O. Box 1968 • Danbury, CT 06813-1968(203) 744-0100 x 210 • Fax: (203) 798-2293

E-mail: dbolton@rarediseases.org

Full payment and registration form must be received by September 12, 2006.

Registration Deadline

NORD Conference Registration FormRoad Map for Rare Disease Research

With the National Institutes of Health (NIH)Office of Rare Diseases

A Conference You Won’t Want to Miss!

Learn about the National Institutesof Health’s Road Map programand how it applies to rare diseases.

Listen to an explanation of the FDA’s Critical Path Initiative,designed to encourage methods to improve the research process.

Attend general sessions that address how rare diseases fit into scientific research beingconducted at the National Institutesof Health

Be a part of a mock InstitutionalReview Board (IRB) meetingto understand the importance of this component of the researchreview process

Learn how the FDA’s Office of Orphan ProductsDevelopment (OOPD)encourages rare diseases research

September 28th–October 1st, 2006Bethesda, MD

2006 NORD Annual Conference

NORD brings to the rare disease

community a unique opportunity to

explore resources, learn from experts,

and network with other individuals who

understand the needs and concerns of this

incredibly large and involved audience.

General sessions address the important research issues

that are of interest to individuals, family members,

colleagues, professionals, and leaders of voluntary health

agencies and support groups. In a cooperative effort

with the Office of Rare Diseases at the National Institutes

of Health (NIH), this year the NORD Annual Conference

offers a special program designed to take advantage of

the close proximity to the NIH and FDA campuses. Join

NORD for an informative and dynamic conference that

gives us hope of new medical progress in the future.

Road Map forRare Disease Research

“... out of the darknessinto the light ...”

6:00 PM to 9:30 PM Conference Check-In/Registration

7:00 PM to 9:00 PM Conference Welcome Reception

7:30 AM Rise & Shine - Coffee with ExhibitorsExhibit Hall open until 5:00 PM

7:30 AM - 8:30 AM Continental Breakfast

8:15 AM - 8:30 AM Welcome AddressAbbey S. Meyers, NORD, President

8:30 AM - 9:15 AM Opening SessionWhat is the NIH Road Map andTranslational Research? Learn about the National Institutes of Health (NIH)Road Map and its role in the promotion of biomedical research. What is translational research?What will be the impact on rare diseases and how can patient organizations be a part of theseadvancements?

9:15 AM - 10:15 AM Panel Discussion“Show Me the Money!” What Is Behind the Funding Process for NIH Research?Representatives from Capitol Hill, the NIH, and patient organizations will talk about thecongressional appropriations process and efforts to increase funding for research. Panelistsinclude Charlene Waldman, Executive Director, Paget Foundation for Paget’s Diseaseof Bone and Related Disorders. Diane Dorman, Vice President of Public Policy for NORD will moderate this session.

10:15AM - 10:45 AM Refreshments in the NORD Exhibitor Hall

10:45 AM - 11: 15 AM General SessionWalking Down the FDA’s Critical Path Dr. Janet Woodcock, FDA Deputy Commissioner for Operations, will explain the agency’snewest initiative to fine-tune the process through which new treatments are developed andbrought to market.

11:15 AM- 11:45 AM General SessionC- Path InitiativeRaymond L. Woosley, MD, PhD, President & CEO, The Critical Path InstituteThe C-Path, formally the Critical Path Institute, is a nonprofit organization whose missionis to “create innovative programs in education and research.” C-Path has three main goals:changing the drug development process to allow faster access to drugs; creating programsto ensure safer drugs and the safer use of drugs; and creating educational programs inadvanced pharmaceutical development.

Friday, September 29, 2006

Thursday, September 28, 2006

11:45 AM - 12:15 PM Critical Path Initiative Panel Drs. Janet Woodcock and Raymond L. WoosleyQuestions and Answers

12:15 PM - 1:30 PM Lunch

1:00 PM - 1:30 PM NORD General Membership Meeting

1:30 PM - 2:15 PM General SessionFDA’s Recent Developments for Orphan Drugsand Humanitarian DevicesDr. Marlene Haffner, Director, FDA, Office of Orphan Products Development.Learn about the latest initiatives at the FDA's Office of Orphan Products Developmentto encourage research on orphan drugs and devices.

2:15 PM - 2:30 PM Open MicWhat are your Questions about Rare Diseases and Research?

2:30 PM - 2:45 PM General SessionDoes the FDA Critical Path Tie into the NIH Road Map?Dr. Andrew Von Eschenbach, Acting FDA CommissionerDr. Von Eschenbach explains the importanceof patients participating in clinical trials andhow the fruits of basic scientific research canbe translated into useable medical productssuch as diagnostics, treatments and cures.

2:45 PM - 3:00 PM Refreshments in the NORDExhibitor Hall

3:00 PM - 4:00 PM Panel DiscussionBehind the Scenes of DrugDevelopment The drug approval process has many layers.This session will look at what happensbehind the scenes in drug development,including the role of FDA Advisory Committees. Panel includes: John Crowley, CEO, Amicus Therapeutics; Ben Lewis, PhD, Senior Director Regulatory Affairs, PrestwickPharmaceuticals; and Dr. Peter Vaccari from the FDA Office of Orphan Products Development.

7:30 AM Rise & Shine - Coffee with ExhibitorsExhibit Hall open until 4:00 PM

7:30 AM - 8:30 AM Continental Breakfast

8:30 AM - 8:45 AM Welcome AddressAbbey S. Meyers, NORD President

Saturday, September 30, 2006

8:45 AM - 10:15 General SessionA Simulated Institutional Review BoardMeeting (IRB): What Do They Really Do?Felix Khin-Maung Gyi, PharmD, CEO, Chesapeake Research Review, Inc.This session is a re-enactment of what takes place during an IRB meeting. A panel of speakers will show what is involved in this important component of the human protection system for those whoparticipate in medical research.

10:15 AM - 10:45 AM Refreshments in the NORD Exhibitor Hall

10:45 AM - 12:00 General SessionThe New Medical Breakthroughs: Real Progress or False Promises?Much has been said about medical advances that promise major improvements and even cures.But, are press reports about gene therapy, stem cell research, and other new technologies based on fact or fiction? A panel of scientists will provide a layman’s update. Speakers willinclude Roscoe O. Brady, MD., Chief NINDS, National Institutes of Health; Dr. Jay Chiorini, Chief, Adeno-associated Virus Biology Unit at NIDCR; Dr. Christopher Austin, Senior Advisor, NIH Chemical Genomics Center, and Dr, Naomi Kleitman, Extramural Research Program, NINDS.

12:00 PM - 1:15 PM Lunch

1:15 PM - 2:00 PM NIH Clinical Center Dr. John Gallin, Director of the NIH Clinical Center (invited), will educate our attendees about this premier national hospital where patients with many rare diseases can seek medicalhelp and participate in research.

2:00 PM - 3:00 PM Panel DiscussionBenefits of Collaboration: Patient Registries and Tissue BanksPatient Registries and Tissue Banks can have an important role in promoting rare diseaseresearch. This discussion will look at several types of registries, their value, and the importanceof privacy and confidentiality agreements with families. The benefits and drawbacks of closed tissue banks will be compared to those of tissue banksthat cover a variety of diseases. Panelists include: Marilyn Baker, Executive Director, Aplastic Anemia & MDSInternational Foundation; Miriam O'Day, Senior Director ofPublic Policy, Alpha-1 Association; Jon Lonsdale PhD,Research Director, National Disease Research Interchange(NDRI); Dr. Carolyn Compton, NIH National Cancer Institute(NCI); and Elisa Eiseman, PhD, RAND.

3:00 PM - 3:15 PM Refreshments in the NORD Exhibitor Hall

3:15 PM - 4:15 PM Panel DiscussionBenefits of Collaboration: Clinical TrialRecruitment Representatives from the NIH Patient Recruitment Program, the FDA, NORD, a pharmaceuticalcompany and a clinical research organization (CRO) will discuss the importance of volunteeringto participate in a clinical trial, what to expect in terms of possible risks and benefits, and whathappens after the trial is finished and the study is published. Panel includes: Ernest Stefanelli,Project Manager, Oncology at PharmaNet; a representative from Amicus Therapeutics; Rino Aldrighetti, President, Pulmonary Hypertension Association; and Dottie Cirelli, Chief, NIH Patient Recruitment Liaison Office.

8:30 AM - 9:30 AM Continental Breakfast

9:30 AM - 9:45 AM Morning AddressMaria Hardin, NORD, Vice President Patient Services

9:45 AM - 11:00 Current Activities and Future Emphasisof the NIH Office of Rare DiseasesAt the request of the congressional House Appropriations Committee to addressthe development of diagnostic tests for rare diseases, the Office of Rare Diseases (ORD)established the Collaboration, Education, and Test Translation (CETT) Program forRare Genetic Diseases. This is a pilot program to promote new genetic test developmentand better understanding of each rare disease. Dr. Stephen Groft, Director of the NIH, ORD, will discuss the important work of this program, along with the latest developments occurring at the ORD.

Sunday October 1, 2006

Welcome Reception Join us on Thursday evening, September 28, 2006, in theHaverford/Baccarett rooms from 7:00 PM to 9:00 PM forcomplimentary refreshments and conversation with fellowattendees.

Exhibitor HallThis is a unique opportunity to be introduced to new and inventive products and services for the special needscommunity. You don't want to miss this display in theHaverford/Baccarett rooms from Thursday at 7:00 PM toSaturday at 4:00 PM. NORD is very grateful to thesecompanies and organizations for supporting this conference.Please be sure to stop by and visit our exhibitors. All refreshment breaks will be held in the Exhibitor Hall.

Rise & ShineIf you are an “early riser” this is the perfect opportunity foryou to have your morning cup(s) of coffee and meet ourexhibitors while checking out their products and services. A great way to start your day! Rise & Shine will be heldFriday and Saturday mornings in the Haverford/Baccarettrooms beginning at 7:30 AM sharp.

General Conference InformationThe NORD Conference will be held at the Hyatt RegencyBethesda located at One Bethesda Metro, Bethesda, MD20814. For the comfort of all attendees, conference meetingrooms, exhibit hall, and common areas are designated asnon-smoking. Casual attire is appropriate for the entireconference.

ScholarshipsA limited number of partial scholarships for conferenceregistration will be made available for NORD members only.These awards are based on financial need and are given on a first-come, first-serve basis. To inquire about theavailability of partial scholarships, please send your writtenor emailed request to the NORD Development Office(dbolton@rarediseases.org) along with your conferenceregistration form. Recipients of these scholarships will becontacted by September 12th, 2006.

Hotel ReservationsA limited number of rooms have been set-aside forconference attendees at the Hyatt Regency Bethesda Hotel,One Bethesda Metro, Bethesda, MD 20814. The specialroom rate for NORD conference attendees is $139 (plus tax) for a single or double. All reservations must be made directly with the Hyatt Regency Bethesda by September 6, 2006 to receive this special room rate. Any reservations made after that date will NOT receivethe conference room rate. Make your reservations online at www.hyattregencybethesda.com, or call (800) 233-1234. When making your reservations pleasemention the NORD Conference to receive the special rate.Please make your reservations early due to limited space. If you wish to share a room with another attendee, contactthe NORD Development Office. NORD will make everyattempt to put you in contact with other attendees who wishto arrange a roommate situation. If you need a roomequipped for people with disabilities, be sure to mention itat the time you make your reservation. There is no chargefor children under 18, when they occupy the same room astheir parents or grandparents.

LocationHyatt Regency BethesdaOne Bethesda Metro Center (7400 Wisconsin Ave), Bethesda, Maryland, USATel: 301-657-1234Fax: 301 657 6453Toll-free: 800-233-1234

Conference Information

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How to RegisterRead the registration form carefully. Please complete theform and return it to NORD by September 12th, 2006.Following that date, registration will only be accepted on a space available basis. Be sure to indicate any of yourspecial needs in the appropriate section of the form. Foreach additional person attending the conference, you mayphotocopy the form and fill it out for each person or contactNORD for additional registration forms. Full payment mustaccompany your form in order to process yourregistration.

Send all forms to: NORD Development OfficeAttn: Donna Bolton-JacksonPO Box 1968Danbury, CT 06813-1968Phone: 203-744-0100 ext 210Fax: 203-798-2293Email: dbolton@rarediseases.org

Hotel Parking Self Park (Overnight): $15.00 All Day: $12.00Valet Park (Overnight): $20.00 All Day: $15.00

Registration FormPlease detach the registration form along the line below, and copy any additional forms for others attending theconference. We want to make your stay at the HyattRegency as pleasurable as possible, so please let us know if you have special needs.

From Washington/National Airport (18 Miles):Take George Washington Parkway to I-495 North, toWisconsin Avenue South, Exit 34 South. Hotel is onright, 2 miles.

From Dulles International (18 miles)Take Dulles Access Road East to I-495, to WisconsinAvenue South, Exit 34 South. Hotel is on right, 2 miles.

From BWI Airport (32 miles):Take I-95 South to I-495, West towards SilverSpring/Chevy Chase. Take 355 South in direction of Bethesda/Washington, DC, which turns to WisconsinAvenue.

Our ultimate goal isto make sure that the

2006 NORD Conferencewill be a memorable

experience for everyone.

Directions