Duchenne Muscular Dystrophy Epidemiology
Jul 22, 2025
Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths; ImCheck’s ICT01 Granted FDA Orphan Drug Designation for Acute Myeloid Leukemia; RedHill Biopharma Gets Positive FDA Feedback on RHB-204 for Crohn’s Disease Approval Pathway; FDA Grants Fast Track Designation to ProMIS Neurosciences’ PMN310 for Alzheimer’s Disease; Oruka Therapeutics Receives IND Clearance for Phase IIa Trial of ORKA-001 in Psoriasis
Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths Sarepta Therapeutics, Inc. has voluntarily and temporarily paused all shipments of ELEVIDYS (delandistrogene moxeparvovec) in the U.S., effective July 22, 2025, following engagement with the FDA on safety labeling u...
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Aug 23, 2024
What Does the Future Hold for Gene Therapy in the Duchenne Muscular Dystrophy (DMD) Treatment Market?
Duchenne Muscular Dystrophy (DMD) is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Gene therapy for Duchenne Muscular Dystrophy (DMD) is poised to revolutionize the treatment landscape by addressing the underlying genetic cause of the...
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Jun 19, 2024
Gene therapy: Following Pfizer’s Unexpected Failure, Sarepta is celebrating its much-awaited full approval! Sarepta’s stock price has shot up over 32%
Duchenne muscular dystrophy (DMD) is a severe genetic disorder primarily affecting young boys between the ages of two and three. As per Delveinsight’s estimates, there were approximately 16K DMD patients in 2023 in the United States, with ˜75% of the patients below the age of 15. This condition leads to progressive...
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