Duchenne Advocacy History

By galvanizing the Duchenne-patient community and bringing our unified voice to Washington, D.C., we have fostered relationships with elected officials who became unwavering Duchenne champions on Capitol Hill, passed banner legislation, and established collaborations with every federal agency that touches Duchenne.

As therapies have moved through development, we’ve told our personal stories to regulators and transformed those stories into quantifiable data. We have catalyzed research funding, and built a regulatory infrastructure for rare disease products that has reshaped the therapeutic, care, and trial environment.

Key Events Timeline

1986Gene producing dystrophin identified
1994PPMD formed by parents and grandparents of persons with Duchenne
2000NIH holds first-ever Duchenne workshop at PPMD’s request
2001Muscular Dystrophy CARE Act (MD-CARE Act) enacted into law by President George W. Bush
2001MD STARnet established by CDC in response to the MD-CARE Act
2005MD Action Plan issued by Muscular Dystrophy Coordinating Committee (MDCC)
2007The Duchenne Registry (previously known as DuchenneConnect) established
2008First Reauthorization of MD-CARE Act
2010CDC’s Duchenne/Becker Care Considerations published in The Lancet Neurology
2011Duchenne “One Voice” Summit hosted by PPMD
2012“Putting Patients First” white paper issued by PPMD calls for regulators to exercise greater flexibility in assessment of novel therapies
2012“Transforming Duchenne Care Initiative” leads to formation of the Certified Duchenne Care Center Program (CDCCP)
2013First formal quantitative study of benefit-risk tradeoffs of Duchenne caregivers
2013PPMD hosts Duchenne Policy Forum with FDA
2014MD CARE Acts Amendments signed into law by President Barack Obama
2014PPMD issues “Patients Are Waiting” collection of patient perspectives on benefit expectations and tolerance for risks and harms
2014Draft guidance on Duchenne, including patient imperatives, submitted to FDA
2014PPMD’s Certified Duchenne Care Center Program certifies its first center
2015PPMD’s Duchenne Drug Discovery Roundtable (DDDR) formed
2015FDA issues draft guidance for Duchenne
2015PPMD forms Adult Advisory Committee (PAAC) to help guide organization’s mission
2016PPMD submits results of preference study on non-skeletal treatment targets to FDA
2016FDA approves EXONDYS 51 (eteplirsen) for treatment of Duchenne
201621st Century Cures signed into law by President Barack Obama; includes Patient Focused Impact Assessment Act, initiated by PPMD
2017FDA approves Emflaza (deflazacort) for treatment of Duchenne
2017 FDA Reauthorization Act signed into law, including patient-focused drug development provisions of PDUFA-VI
2017DDDR conducts series of meetings covering clinical trial harmonization, clinical trial optimization, and clinical trial harmonization
2017PPMD hosts meetings on the role of inflammation, cardiac outcome measures in Duchenne, and the burden of clinical trial participation.
2017PPMD launches global study of benefit-risk preferences and a study focused on risk tolerance in gene therapy interventions
2018FDA issues final guidance for Duchenne
2018PPMD leads the refinement & implementation of ICD-10 code for Duchenne/Becker
2018Updated Duchenne/Becker Care Considerations published in The Lancet Neurology
2018PPMD hosts Compass Patient-Focused Drug Development Meeting
2018PPMD launches global study of benefit-risk preferences on Gene therapy
2019PPMD hosts second meeting on role of inflammation with FDA, NIH and researchers
2019PPMD publishes The Duchenne Registry 10 Year Report
2019FDA approves VYONDYS 53 for treatment of Duchenne patients amendable to exon 53 skipping
2020FDA approves VILTEPSO for treatment of Duchenne patients amendable to exon 53 skipping
2021PPMD completes Newborn Screening Pilot Program in New York state
2021PPMD initiatives process for updating the Community-led Guidance for FDA
2021The BENEFIT Act is introduced in House and Senate
2021FDA approves Casimersen for the treatment of Duchenne patients amenable to exon 45 skipping