Regenxbio filed a lawsuit in 2020 alleging that Elevidys infringed a gene-therapy patent that the company licenses from ...

Lee, 34 years old, from Suwon, Gyeonggi Province, watched her father collapse from a cerebral hemorrhage and undergo surgery in 2021. He spent six months in a university hospital and a rehabilitation ...

Eric Dane died on Thursday, Feb. 19, less than a year after he was diagnosed with ALS. The life expectancy for many is three to five years after diagnosis; however, some can live longer, according to ...

Dyne Therapeutics Inc. (NASDAQ: DYN) is one of the 13 hot stocks to buy with the highest upside potential. As of February 13, ...

Del-desiran (also known as AOC1001) is an antibody-oligonucleotide conjugate comprised of a DMPK small interfering RNA (siRNA ...

A preclinical study led by the Germans Trias i Pujol Research Institute (IGTP), in collaboration with the Institut de ...

Sarepta Therapeutics has provided additional safety data on its gene therapy Elevidys as a Duchenne muscular dystrophy patient group petitions the FDA to update the medicine’s label. Altogether, eight ...

Sarepta Therapeutics Inc. investors finally got some good news, but analysts worry that it could still take a while for doctors and patients to again feel comfortable using the biotechnology company’s ...

Stock ends down 36% amid FDA scrutiny on Sarepta gene therapies Analysts warn of damage to management credibility Company loses platform technology designation July 18 (Reuters) - U.S. regulators ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...