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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 […]
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Pfizer Shares Update on Phase 3 Study of Investigational Mini-Dystrophin Gene Therapy
PPMD is deeply disappointed to share that Pfizer Inc. announced today that CIFFREO, a Phase […]
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Pitolisant found to reduce fatigue, daytime sleepiness in DM1 patients
After nearly three months of treatment with pitolisant, adults with myotonic dystrophy type 1 (DM1) […]
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While we’re all on unique paths, I welcome input from my community
Over the past couple months, I’ve shared my decision to try physical therapy in an […]
