Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated ...

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...

A patient died from liver injury while taking the gene therapy Elevidys Sarepta says more than 800 patients have received the ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

Entrada Therapeutics Inc. has received authorization from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) and research ethics committee to initiate a phase I/II study of ENTR-601 ...

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...