Entrada Therapeutics has secured authorisation from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and ...

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

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

Shares of Sarepta Therapeutics SRPT plunged nearly 24% last week after the company reported the death of a patient following ...

Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

CRISPR technique can target single genes essential for cancer cell survival, toppling the whole structure, says nanomedicine ...

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 money is developing treatments for diabetes, cancer and Alzheimer's disease. It could also mean a cure for a Ridgefield ...

Sarepta stock may recover sharply as additional information and data reveal that the adverse event reported by the company is ...

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

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