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Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above
See the previous update from June 22, 2023 here FDA Grants Traditional Approval for Ambulatory […]
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PAAC Travel Tips
Anyone living with Duchenne muscular dystrophy knows that there are extra steps to EVERYTHING. Traveling […]
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FDA Expands ELEVIDYS Label Indication for Ages 4 Years and Older
PPMD is excited to share that the FDA has made the decision to expand the […]
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Cranbury Pharmaceuticals Receives U.S. FDA Approval for First Generic Version of Emflaza® Oral Suspension (deflazacort) for Duchenne Muscular Dystrophy
The FDA approved the first generic version of Emflaza® for treating Duchenne in the US. […]
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Phase 3 trial of Pfizer DMD gene therapy fails to meet its goals
The gene therapy developed by Pfizer called fordadistrogene movaparvovec failed to significantly improve motor function […]
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Newborn Screening for Duchenne: PPMD’s Collaborative Future Planning Efforts
On Saturday, June 8th, PPMD convened a vital meeting of physicians, physical therapists, genetic counselors, […]
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FDA approves first generic of Emflaza oral suspension for DMD
The U.S. Food and Drug Administration (FDA) has approved the first generic version of Emflaza […]
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I am always learning more about life with Duchenne
When three of my sons — Max, 18, Rowen, 15, and Charlie, 13 — were […]
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Build Your Own Care Binder at PPMD’s 30th Annual Conference
PPMD is excited to share that at our 30th Annual Conference, taking place June 27-29, […]
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Cranbury Pharmaceuticals (Tris Pharma) Receives FDA Approval for First Generic Version of Emflaza® (deflazacort) for Duchenne
Cranbury Pharmaceuticals, a subsidiary of Tris Pharma, today announced the U.S. Food and Drug Administration […]
