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
| 1986 | Gene producing dystrophin identified |
| 1994 | PPMD formed by parents and grandparents of persons with Duchenne |
| 2000 | NIH holds first-ever Duchenne workshop at PPMD’s request |
| 2001 | Muscular Dystrophy CARE Act (MD-CARE Act) enacted into law by President George W. Bush |
| 2001 | MD STARnet established by CDC in response to the MD-CARE Act |
| 2005 | MD Action Plan issued by Muscular Dystrophy Coordinating Committee (MDCC) |
| 2007 | The Duchenne Registry (previously known as DuchenneConnect) established |
| 2008 | First Reauthorization of MD-CARE Act |
| 2010 | CDC’s Duchenne/Becker Care Considerations published in The Lancet Neurology |
| 2011 | Duchenne “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) |
| 2013 | First formal quantitative study of benefit-risk tradeoffs of Duchenne caregivers |
| 2013 | PPMD hosts Duchenne Policy Forum with FDA |
| 2014 | MD CARE Acts Amendments signed into law by President Barack Obama |
| 2014 | PPMD issues “Patients Are Waiting” collection of patient perspectives on benefit expectations and tolerance for risks and harms |
| 2014 | Draft guidance on Duchenne, including patient imperatives, submitted to FDA |
| 2014 | PPMD’s Certified Duchenne Care Center Program certifies its first center |
| 2015 | PPMD’s Duchenne Drug Discovery Roundtable (DDDR) formed |
| 2015 | FDA issues draft guidance for Duchenne |
| 2015 | PPMD forms Adult Advisory Committee (PAAC) to help guide organization’s mission |
| 2016 | PPMD submits results of preference study on non-skeletal treatment targets to FDA |
| 2016 | FDA approves EXONDYS 51 (eteplirsen) for treatment of Duchenne |
| 2016 | 21st Century Cures signed into law by President Barack Obama; includes Patient Focused Impact Assessment Act, initiated by PPMD |
| 2017 | FDA approves Emflaza (deflazacort) for treatment of Duchenne |
| 2017 | FDA Reauthorization Act signed into law, including patient-focused drug development provisions of PDUFA-VI |
| 2017 | DDDR conducts series of meetings covering clinical trial harmonization, clinical trial optimization, and clinical trial harmonization |
| 2017 | PPMD hosts meetings on the role of inflammation, cardiac outcome measures in Duchenne, and the burden of clinical trial participation. |
| 2017 | PPMD launches global study of benefit-risk preferences and a study focused on risk tolerance in gene therapy interventions |
| 2018 | FDA issues final guidance for Duchenne |
| 2018 | PPMD leads the refinement & implementation of ICD-10 code for Duchenne/Becker |
| 2018 | Updated Duchenne/Becker Care Considerations published in The Lancet Neurology |
| 2018 | PPMD hosts Compass Patient-Focused Drug Development Meeting |
| 2018 | PPMD launches global study of benefit-risk preferences on Gene therapy |
| 2019 | PPMD hosts second meeting on role of inflammation with FDA, NIH and researchers |
| 2019 | PPMD publishes The Duchenne Registry 10 Year Report |
| 2019 | FDA approves VYONDYS 53 for treatment of Duchenne patients amendable to exon 53 skipping |
| 2020 | FDA approves VILTEPSO for treatment of Duchenne patients amendable to exon 53 skipping |
| 2021 | PPMD completes Newborn Screening Pilot Program in New York state |
| 2021 | PPMD initiatives process for updating the Community-led Guidance for FDA |
| 2021 | The BENEFIT Act is introduced in House and Senate |
| 2021 | FDA approves Casimersen for the treatment of Duchenne patients amenable to exon 45 skipping |
