Inherited repeat mutations in the HTT gene expand in neurons over decades ... This can happen both in cells of the germ line (which form sperm and egg cells) and in somatic cells (cells of the ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Scientists at the Allen Institute have made a major breakthrough in research for people living with Dravet Syndrome, which is ...
The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials. The series C was led by Arch Venture Partners and TCGX, with new investors QIA ...
One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...
An Indian-developed gene therapy for certain blood cancers has shown a 73 per cent response rate among patients in India. The results of the clinical trials were published in The Lancet ...
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