PPMD’s End Duchenne [Remote] Tour: Long Island, NY

#EndDuchenne

Agenda10:00 am – 10:05 am Welcome Ryan Fischer

Parent Project Muscular Dystrophy10:05 am – 10:20 am Who is PPMD? Ryan Fischer

Parent Project Muscular Dystrophy

10:20 am – 10:30 am Get InvolvedPPMD’s Connect: Long IslandRace to End Duchenne: TBD

Dianne CarrollParent, PPMD Connect Coordinator

10:30 am – 10:40 am Hope for Javier Jen PortnoyParent, Founder

10:40 am – 11:10 pm Genetics, The Duchenne Registry and Your FamilyUnderstanding Your MutationDecode DuchenneThe Duchenne Registry

Niki Armstrong, MS, CGCParent Project Muscular Dystrophy

11:10 am – 12:30 pm Duchenne Expert Panel: Research and CareCardiology Standards of CareNeuromuscular Standards of CarePhysical TherapyCare Coordination and Local Resources

Moderator: Ryan FischerParent Project Muscular DystrophyPeter Morelli, MDStony Brook Children’sYaacov Anziska, MDSUNY DownstateChristina Rao, PTYale-New Haven Children's HospitalDawn Dawson, CPNP/ANPStony Brook Children’s

Who is PPMD?

Ryan FischerSVP, Community Engagement

#EndDuchenne

• What was the current state of research & care in Duchenne?

• What was the federal government investment in Duchenne?

• What data existed about the Duchenne population?

• What are the gaps we need to fill in order to move the needle toward better care & treatments for Duchenne?

Critical Missing Pieces

Data

Federal

Dollars

Outcome

Measures

Industry NaturalHistory

CareStandardsAdvocacy

1994 Questions

#EndDuchenne

Impact on Research

invested by PPMD into Duchenne

research & therapy to date

awarded, supporting nearly every major

therapeutic approach

in Duchenne at any given time

with additional promising

therapies in development

$50 million+ Over 260 research grants

~35 clinical trials 3 U.S.drug approvals

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March 6, 2020

Duchenne Drug Development PipelineThe Drug Development Pipeline is full of potential treatments that are being tested. These include therapeutic approaches that restore or replace dystrophin and those that treat Duchenne symptoms. The goal? To test combinations of these therapies to create the best “cocktail” for each patient.

Restoring or Replacing Dystrophin

Combating Fibrosis

Reducing Inflammation

Regulating Calcium Balance

Improving Muscle Growth & Protection

Restoring the Cells Energy

Improving Heart

Function

The Drug Development Pipeline is full of potential treatments that are being tested. These include therapeutic approaches that restore or replace dystrophin and those that treat Duchenne symptoms. The goal? To test combinations of these therapies to create the best “cocktail” for each patient.

DUCHENNE DRUGDEVELOPMENT PIPELINE

March 9, 2020

#EndDuchenne

APPROVAL

BROADLY ENGAGING THEDEVELOPMENT PIPELINE

For over two decades, Parent Project Muscular Dystrophy (PPMD) has contributed to each stage of the drug development pipeline, awarding grants, filling in critical gaps, convening stakeholders, and redefining the clinical trial landscape.

Exploratory research awards

Validation & replication study services

Updated Duchenne Care Consideration Guidelines & Family Guide

Duchenne Newborn Screening Program

The Duchenne Registry ChildMuscleWeakness.org

– an early diagnosis program AAP motor delay tool ICD-10 code refinement

Clinical trial support Drug development

research awards FDA & regulatory

engagement The Duchenne Registry trial

recruitment services Multichannel community

outreach & education series Clinical trial participant

education Expert consultation

informing trial enrollment & design

Certified Duchenne Care Center program & Clinical Trial Awareness program

Duchenne Specialty Care Workshops

PPMD / C-Path Duchenne Regulatory Science Consortium

Duchenne Drug Development Roundtable – engaging sponsors in pre-competitive space

Partnering with federal agencies (MDCC, FDA, CDC, NIH, DoD, CMS, SSA)

The Duchenne Registry trial readiness services

Duchenne FDA Guidance for industry

Trial education and recruitment

Duchenne community engagement

Leading creation of forward thinking expert publications, i.e.: Putting Patients First: Patients are Waiting, & numerous patient & caregiver preference study publications

Advisory Committee & IND meeting support

Leading passage of 5 federal bills, securing Duchenne-specific federal funding, & supporting rare disease legislation

Pioneering access, coverage, & reimbursement strategy

Decode Duchenne, free genetic testing

Patient engagement initiatives

Post-marketing strategy development

Payer engagement

PRECLINICALDISCOVERY &

PHASE 1TRIAL READINESS/ REGULATORY

& RECRUITMENTPHASE 2/3

& ACCESSPOST-MARKET

#EndDuchenne

Impact on Advocacy

signed into law, reshaping the

Duchenne landscape

sent to Members of Congress

in federal funding leveraged by PPMD

community into research

face-to-face with Members of Congress

4 bills 12,000 messages &action alerts

Over $600 million More than 2,000meetings

#EndDuchenne

• Sign up to become an advocate to receive action alerts in your inbox at ParentProjectMD.org/AdvocacyAlerts

• PPMD can assist in organizing in district meetings with your local representatives

• Contribute to our preference studies and represent our community at meetings with federal agencies

• Adults living with Duchenne were the first patient representatives to serve on an FDA Advisory Committee for FDA decisions

• Attend PPMD’s Advocacy Conference– Held Q1 each year in Washington, DC - scheduled visits on Capitol Hill

are arranged for all attendees to meet with the offices of their congressional members and tell their stories

How YOU Can Advocate

#EndDuchenne

Impact on Care

added to average lifespan due to PPMD-

led advances

awarded certification by PPMD across the US as of January 2020

in PPMD network of certified clinics

of identifying & addressing gaps in care through

specialty workshops & consensus meetings

10 years 27 clinics 700 care providers 20 years

#EndDuchenne

• In partnership with the CDC

• First published in January 2010, with updated guidelines published in 2018 in Lancet Neurology

• Diagnosis and management of Duchenne muscular dystrophy (3 parts):

– Part 1: Diagnosis, neuromuscular, rehabilitation, endocrine, & gastrointestinal and nutritional management

– Part 2: Respiratory, cardiac, bone health, & orthopedic management

– Part 3: Primary care, emergency management, psychosocial care, & transitions of care across the lifespan

Duchenne Care Considerations• Led to the development of the Certified

Duchenne Care Center program (2014)– Care Considerations = requirements for

certification– Certification requirements were adapted for

adult certification, with the first adult CDCC certified in 2019

– Expanded program globally to include 2 international centers

• PPMD also led the effort to an ICD-10 code approval specifically for Duchenne/Becker to

– Goal: improve longitudinal surveillance of data

*As of January 2020

SEATTLE CHILDREN’S HOSPITAL

UCSF

UC DAVIS

UCLA

STANFORD CHILDREN’S HOSPITAL

CHILDREN’S HOSPITAL L.A.

PRIMARY CHILDREN’S HOSPITAL

CHILDREN’S HOSPITAL COLORADO

UVA

NATIONWIDE CHILDREN’S HOSPITAL

DUKE UNIVERSITY MEDICAL CENTER

KENNEDY KRIEGER INSTITUTE

CHILDREN’S HOSPITAL OF THE KING’S DAUGHTERS

CHILDREN’S NATIONAL HOSPITAL

YALE NEW HAVEN CHILDREN’S HOSPITAL

CHILDREN’S HOSPITAL WISCONSIN

LURIE CHILDREN’S HOSPITAL

GILLETTE CHILDREN’S HOSPITAL AMERICAN FAMILY CHILDREN’S HOSPITAL UNIVERSITY OF ROCHESTER

NEMOURS/ALFRED I. DUPONT HOSPITAL FOR CHILDREN

CHILDREN’S MERCY HOSPITAL

CINCINNATI CHILDREN’S HOSPITAL

UNIVERSITY OF IOWA CHILDREN’S HOSPITAL

ARKANSAS CHILDREN’S HOSPITAL

UNIVERSITY OF MISSOURI

CHILDREN’S HOSPITAL OF RICHMOND AT VCU

Plus, two Global Certified Duchenne Care Centers: Red Cross War Memorial Children’s Hospital (South Africa) and Motol University Hospital in (Czech Republic)

PPMD’S CERTIFIED DUCHENNE CARE CENTER PROGRAM

27 CENTERS across the

United States

700 CARE PROVIDERS

caring for individuals

with Duchenne

3,300 PATIENTS

served at certified centers

79 CLINICAL TRIALS located at

certified centers

+1,150 TRIAL PARTICIPANTS

currently enrolled or in

active recruitment

*As of January 2020

SEATTLE CHILDREN’S HOSPITAL

UCSF

UC DAVIS

UCLA

STANFORD CHILDREN’S HOSPITAL

CHILDREN’S HOSPITAL L.A.

PRIMARY CHILDREN’S HOSPITAL

CHILDREN’S HOSPITAL COLORADO

UVA

NATIONWIDE CHILDREN’S HOSPITAL

DUKE UNIVERSITY MEDICAL CENTER

KENNEDY KRIEGER INSTITUTE

CHILDREN’S HOSPITAL OF THE KING’S DAUGHTERS

CHILDREN’S NATIONAL HOSPITAL

YALE NEW HAVEN CHILDREN’S HOSPITAL

CHILDREN’S HOSPITAL WISCONSIN

LURIE CHILDREN’S HOSPITAL

GILLETTE CHILDREN’S HOSPITAL AMERICAN FAMILY CHILDREN’S HOSPITAL UNIVERSITY OF ROCHESTER

NEMOURS/ALFRED I. DUPONT HOSPITAL FOR CHILDREN

CHILDREN’S MERCY HOSPITAL

CINCINNATI CHILDREN’S HOSPITAL

UNIVERSITY OF IOWA CHILDREN’S HOSPITAL

ARKANSAS CHILDREN’S HOSPITAL

UNIVERSITY OF MISSOURI

CHILDREN’S HOSPITAL OF RICHMOND AT VCU

Plus, two Global Certified Duchenne Care Centers: Red Cross War Memorial Children’s Hospital (South Africa) and Motol University Hospital in (Czech Republic)

CDCCC_US_letter_1pp_Nov_25.indd 1CDCCC_US_letter_1pp_Nov_25.indd 1 1/6/20 10:16 AM1/6/20 10:16 AM

#EndDuchenne

Engagement in the Community

across the United States, providing local

families outreach & mentoring

across the globe have registered in The

Duchenne Registry since launch

raised through Race to End Duchenne & family-led grassroots

events since 1994

reached in person, through 25 Annual Conferences, End

Duchenne Tour stops, Roundtable discussions, & Advocacy Conferences

26 PPMD Connect locations

Over 5,000 patients & carriers

$36 million+ 14,310 families

#EndDuchenne

• Coach To Cure MD, presented by Werner Ladder, is a partnership between the American Football Coaches Association (AFCA) and PPMD

• This year on September 26, 2020 coaches nationwide will wear a Coach to Cure MD patch and mention the program in interviews

• Know a team who would be interested? Interested in attending a game near you?

• CoachToCureMD.org

New York teams who have participated:

• Hamilton College• Rensselaer Polytech Institute• Morrisville State• Hobart College• The College at Brockport• St. John Fischer College• Pace University• Marist College Football • Buffalo State College• Syracuse University Football• Utica College

Coach To Cure MD

#EndDuchenne

• PPMD will be hosting our annual conference virtually July 22-25– The PPMD team is committed to making this conference as complete and robust

as our in-person meeting including panels about research, care & advocacy – even opportunities for virtual meet-ups with other families!

– For updates, please visit: ParentProjectMD.org/Conference• We will also be hosting a virtual newly diagnosed track starting the week of June

22nd with a virtual meet & greet with other families, Pat Furlong, and other PPMD staff members followed by a ”Duchenne 101” webinar the week of July 9th

– To register for this track, please visit: join.parentprojectmd.org/newlydiagnosedscholarship

PPMD’s 2020 Annual Conference

#EndDuchenne

Other Ways to Get Involved

Race to End Duchenne

Find a local PPMD Connect

group

Local DIY Events

Hope For JavierJen Portnoy

hopeforjavier.orginfo@hopeforjavier.org

Comprehensive Duchenne Care

#EndDuchenne

• Duchenne Family Guide– Partnered with MDA, Treat-NMD, and WDO– Includes the Duchenne Care Considerations in an

“easier to digest” format• New Diagnosis and Early Care Guide

– Introduction to Duchenne/Becker and genetic testing, adjusting to the diagnosis and support, early care, and introduction to clinical trials

• Imperatives for Healthcare Providers– One-page fact sheets for healthcare providers unfamiliar

with Duchenne– Versions for pediatric and adult patients– Also available in Spanish

• Education Matters– Comprehensive guides for parents and teachers

Care Guides

#EndDuchenne

• PPMD App– Includes Duchenne emergency information

• PPMD emergency information cards– Also available in Spanish

• PPMD large weatherproof emergency information cards for wheelchairs, scooters, and backpacks

• PJ Nicholoff Steroid Protocol– Guide for families and healthcare providers to navigate

steroid stress dosing, tapering, and what to do in the case of a missed dose

• Online Resources– COVID-19 Resource Center– Emergency care and hospital checklists– Fracture management

Emergency Care Information

Duchenne Expert Panel

Cardiology Care Peter Morelli, MDStony Brook Children’s

Neuromuscular Care Yaacov Anziska, MDSUNY Downstate

Physical Therapy Christina Rao, PTYale-New Haven Children’s Hospital

Care Coordination & Local Resources

Dawn Dawson, CPNP/ANPStony Brook Children’s

Genetics Niki Armstrong, MS, CGCParent Project Muscular Dystrophy

Duchenne Expert Panel

Please submit your questions through the chat box

Cardiology Care Peter Morelli, MDStony Brook Children’s

Neuromuscular Care Yaacov Anziska, MDSUNY Downstate

Physical Therapy Christina Rao, PTYale-New Haven Children’s Hospital

Care Coordination & Local Resources

Dawn Dawson, CPNP/ANPStony Brook Children’s

Genetics Niki Armstrong, MS, CGCParent Project Muscular Dystrophy

Thank you!

@ParentProjectMD

ENDDUCHENNE.ORG