Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

Marcos Reyes and Valeria Martínez are in a tough spot, unsure which of their twins should receive the treatment they ...

Just a few weeks after closing a $155 million series B, RNA editing biotech AIRNA is bringing an experienced gene therapy ...

Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of limb-girdle muscular dystrophy (LGMD), a group of rare genetic disorders marked ...

The law firm of Kessler Topaz Meltzer & Check, LLP ( is currently investigating potential violations of the federal ...

Sarepta Therapeutics (NASDAQ:SRPT) stock draws an Overweight rating at Wells Fargo, based on commercial prospects for ...

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 ...

The findings suggest that the body's own immune system may be undermining the treatment with gene therapy such as Elevidys ...

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute ...

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Sarepta and Roche announced a 16-year-old US patient dosed with Elevidys died of acute liver failure in March 2025.