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Pfizer’s Phase 3 Trial for Gene Therapy in Boys with Duchenne Muscular Dystrophy Fails to Show Functional Improvement

Availability of new drugs result in U.S. Task Force’s consideration of routine kidney disease screening

Sanofi gets FDA approval for Xenpozyme- first ever drug to treat Acid Sphingomyelinase Disease

Must Read

J&J Reports Positive Phase 2/3 Results for Imaavy in Warm Autoimmune Hemolytic Anemia

Edgewise Reports Phase 2 CIRRUS-HCM Results for EDG-7500 Ahead of Planned Phase 3 Development

ARPA-H Awards Up to $160 Million for Personalized Genetic Medicine Development

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