Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...

Childhood-onset striatonigral neurodegeneration robs children of the ability to walk and talk by age five. Researchers at ...

An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

A comprehensive approach, including robust preclinical research and a streamlined regulatory process, is the key ...

The arc of biotechnology is one of breakthroughs and struggles, writes Tim Hunt, CEO of the Alliance for Regenerative ...

Congenital hearing loss refers to impaired auditory function that occurs due to genetic causes. GJB2 is the gene responsible ...

The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham ...

The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.

Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...

R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...