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 ...
Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...
The arc of biotechnology is one of breakthroughs and struggles, writes Tim Hunt, CEO of the Alliance for Regenerative ...
Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...
Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
With a storied history in vaccine development, India's Bharat Biotech is shaking things up and answering the call of advanced ...
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
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