This year marks 20 years since passage of the MD-CARE Act, landmark legislation led by PPMD that ushered in a new era of federal investment into the muscular dystrophies. This year also marked the first time ever PPMD organized an all-virtual Advocacy Conference.

It was a record-breaking year for participation, with over 425 advocates participating from 43 states in over 300 virtual meetings. We also had over 1,000 action alerts sent to Congress from community members across the country while advocates participated in meetings.

Record breaking participation has translated into record breaking results! We are pleased to report a very successful outcome for the Duchenne Fiscal Year 2022 (FY22) funding letters signatures.

122 members of Congress in total (94 in the House and 28 in the Senate) agreed to support our request sending a strong message of support behind Duchenne research, public health, drug development, and patient support initiatives. Both letters have bi-partisan support, which is critical for these efforts.

PPMD is grateful to the Foundation to Eradicate Duchenne for their partnership and collaboration on the Advocacy Conference.

WHAT WAS IN THE REQUEST TO CONGRESS?

CENTERS FOR DISEASE CONTROL

• Asks Congress to increase funding for CDC’s Muscular Dystrophy Program to $8 million and that money be used to evaluate:

• • The impact of the Duchenne Care Considerations, including on rural & underserved areas.
  • Availability of consistent and coordinated care for adults with Duchenne.
  • Impact of Duchenne and Becker on mental health of patients and caregivers.

• It also requests that CDC issue a report on program priorities including progress to date and priorities for upcoming years.

NATIONAL INSTITUTES OF HEALTH

• Requests the NIH to focus Duchenne research on:
  • Challenges related to gene therapies, including delivery to individuals with neutralizing antibodies, manufacturing, and redosing of gene therapy.
  • The impact of Duchenne on both the brain and the heart.

FOOD AND DRUG ADMINISTRATION

• Encourages the FDA to update the 2018 FDA Guidance on Duchenne to reflect advancement in gene therapies.
• Convene a meeting to evaluate how to minimize the use of placebo in clinical trials.

DEPARTMENT OF DEFENSE

• Requests an increase from $10 million $12 million for the Duchenne Muscular Dystrophy Research Program (DMDRP) within the Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP).

Download the Letter >

House Signatures

House Member	State and District
Terri A. Sewell	AL-7
Mark DeSaulnier	CA-11
Julia Brownley	CA-26
Ted W.	CA-33
Lucille Roybal-Allard	CA-40
Mark Takano	CA-41
Nanette Diaz Barragán	CA-44
Sara Jacobs	CA-53
Doris Matsui	CA-6
Ami Bera, M.D.	CA-7
Joe Neguse	CO-2
Jason Crow	CO-6
John B. Larson	CT-1
Joe Courtney	CT-2
Jim Himes	CT-4
Jahana Hayes	CT-5
Eleanor Holmes Norton	DC at large
Lisa Blunt Rochester	DE
Gus M. Bilirakis	FL-12
Alcee L. Hastings	FL-20
Ted Deutch	FL-22
Michael Waltz	FL-6
Stephanie Murphy	FL-7
Bill Posey	FL-8
Nikema Williams	GA-5
Lucy McBath	GA-6
Carolyn Bourdeaux	GA-7
Bill Foster	IL-11
Sean Casten	IL-6
Danny K. Davis	IL-7
Raja Krishnamoorthi	IL-8
Jan Schakowsky	IL-9
André Carson	IN-7
John Yarmuth	KY-3
James P. McGovern	MA-2
Seth Moulton	MA-6
Stephen F. Lynch	MA-8
Jamie Raskin	MD-8
Chellie Pingree	ME-1
Jared Golden	ME-2
Haley Stevens	MI-11
Debbie Dingell	MI-12
Rashida Tlaib	MI-13
Peter Meijer	MI-3
Daniel T. Kildee	MI-5
Elissa Slotkin	MI-8
Angie Craig	MN-2
Emanuel Cleaver, II	MO-5
Michael Guest	MS-3
Donald Norcross	NJ-1
Donald M. Payne, Jr.	NJ-10
Mikie Sherrill	NJ-11
Josh Gottheimer	NJ-5
Tom Malinowski	NJ-7
Albio Sires	NJ-8
Bill Pascrell, Jr.	NJ-9
Lee Zeldin	NY-1
Jerrold Nadler	NY-10
Nicole Malliotakis	NY-11
Carolyn B. Maloney	NY-12
Antonio Delgado	NY-19
Andrew R. Garbarino	NY-2
Elise M. Stefanik	NY-21
Claudia Tenney	NY-22
John Katko	NY-24
Joseph D. Morelle	NY-25
Brian Higgins	NY-26
Chris Jacobs	NY-27
Thomas R. Suozzi	NY-3
Kathleen M. Rice	NY-4
Troy Balderson	OH-12
Steve Stivers	OH-15
Bill Johnson	OH-6
Peter A. DeFazio	OR-4
Brian Fitzpatrick	PA-1
Fred Keller	PA-12
Conor Lamb	PA-17
Dwight Evan	PA-3
Mary Gay Scanlon	PA-5
Susan Wild	PA-7
John Rose	TN-6
David Kustoff	TN-8
Steve Cohen	TN-9
Sheila Jackson Lee	TX-18
Eddie Bernice Johnson	TX-30
Colin Allred	TX-32
Lizzie Fletcher	TX-7
Al Green	TX-9
Gerald E. Connolly	VA-11
Abigail D. Spanberger	VA-7
Peter Welch	VT at large
Marilyn Strickland	WA-10
Adam Smith	WA-9
Carol D. Miller	WV-3

Senate Signatures

Member First	Member Last	State
Mark	Kelly	AZ
Kyrsten	Sinema	AZ
Diane	Feinstein	CA
Alex	Padilla	CA
Michael	Bennet	CO
Richard	Blumenthal	CT
Chris	Coons	DE
Rick	Scott	FL
Raphael	Warnock	GA
Dick	Durbin	IL
Ed	Markey	MA
Elizabeth	Warren	MA
Chris	Van Hollen	MD
Susan	Collins	ME
Angus	King	ME
Gary	Peters	MI
Debbie	Stabenow	MI
Amy	Klobuchar	MN
Roger	Wicker	MS
Kevin	Cramer	ND
Jeanne	Shaheen	NH
Cory	Booker	NJ
Bob	Menendez	NJ
Catherine	Cortez Mastro	NV
Jacky	Rosen	NV
Kirsten	Gillibrand	NY
Sherrod	Brown	OH
Tammy	Baldwin	WI

WHAT HAPPENS NEXT?

The timing for when the funding bill (known as appropriations) is finalized can often be unpredictable and can be delayed for many reasons – rest assured, they eventually get done. The good news is you have done the hard part already, ensuring the best chance for Duchenne related report language and funding are included for agencies that impact Duchenne in the coming year. We will keep you posted when the bills pass through and are signed into law.

Thank you for all your efforts!

Save the Date for next year’s in-person Advocacy Conference

The 2022 Advocacy Conference is planned to be in person next year March 6-8, 2022. We hope to see you in Washington D.C.! Sign up for PPMD emails to be notified when registration is open.