Sarepta Therapeutics Halts ELEVIDYS Shipments for Non-Ambulatory DMD Patients

Sarepta Therapeutics Inc. (NASDAQ:SRPT) is one of the best low priced pharma stocks to buy now. Towards the end of June, Sarepta Therapeutics started facing a temporary suspension of shipments for ELEVIDYS, which is its gene therapy for Duchenne muscular dystrophy/DMD, specifically for non-ambulatory patients. This decision was announced around June 16 and followed two reported cases of fatal acute liver failure in non-ambulatory patients who received the treatment.

The most recent death involved a 15-year-old patient. These severe side effects have led Sarepta to pause the ENVISION Phase 3 confirmatory trial (SRP-9001-303), which includes both older ambulatory and non-ambulatory individuals, to allow for protocol updates and regulatory discussions.

DMD primarily affects males, with symptoms typically appearing between the ages of 2 and 3. It progresses rapidly and leads to the gradual loss of muscle function in the skeletal, heart, and lung muscles. DMD is caused by a mutation in the DMD gene, which is responsible for producing dystrophin, a protein vital for muscle cell integrity.

Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company that discovers and develops RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases.

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Disclosure: None. This article is originally published at Insider Monkey.