Eisa Hussain was born with a condition meaning his body could not fight off infections and he was expected to die before the ...

Eisa Hussain was born with a severe form of leukocyte adhesion deficiency 1 (LAD-1), which can carry a "death sentence" if ...

A boy with an ultra-rare immune disease which can carry a “death sentence” is living a normal life after taking part in a ...

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 in Gene Therapy suggests the body’s natural immune system might be ...

Solid Bio said recently it has started dosing additional patients in its phase 1/2 trial of SGT-001 – called IGNITE DMD – and will have further results from the first three patients treated ...

Martin Mwita, 20, is the first patient in St. Louis and among the first across the country to receive a new gene therapy for ...

following the death of a 16-year-old patient in the U.S., Sarepta and Roche have temporarily halted three clinical trials in Europe of their Duchenne muscular dystrophy (DMD) gene therapy Elevidys.

The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to pause dosing in another study.

In March, Sarepta Therapeutics, Inc. SRPT shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy ... especially after the recent patient death due to ...

In March, Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients ... half of 2025—especially after the ...