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Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion 
Upcoming Webinars: Spotlighting Federally Funded Research Studies in Duchenne 
FDA Approval of AMONDYS 45 (casimersen) 
FDA Approves Sarepta Therapeutics’ Amondys 45 for Treatment of DMD Amenable to Skipping Exon 45 
FDA Grants Accelerated Approval to AMONDYS 45™ (Casimersen) 
Exon Skipping Dominates DMD Treatment, But What’s on Horizon? 
FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation 
Sarepta Therapeutics Announces FDA Approval of AMONDYS 45™ (casimersen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 45 
Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Individuals With Generalized Myasthenia Gravis 
Senate Duchenne Champions Reintroduce BENEFIT Act Heading into PPMD’s Virtual Advocacy Conference 
Golf Outings: The Perfect Socially-Distanced DIY Event! 
Italfarmaco provides an update on Givinostat 
Givinostat Continues to Delay DMD Progression in Long-term Study, New Data Show 
Rare Disease Day at NIH, Set for March 1, Growing Year by Year 
Golf Tournaments: The Perfect Socially-Distanced DIY Event! 
Italfarmaco Community Letter – February 2021 
Introduction to Edgewise Therapeutics 
Embrace This Moment – Safety and Emergency Care Webinar 
MDA Welcomes Dr. Don Wood as its New President and CEO 
CureDuchenne Webinar – An Introduction to REGENXBIO and RGX-202 
Rare disease day 2021 – La Force DMD 
How Expedited Drug Approval Impacts the Neuromuscular Disease Treatment Landscape 
MDA’s 2021 Advocacy Plan