New gene therapy cures 1st New Yorker of sickle cell anemia
Mía had dystrophic epidermolysis bullosa, better known as butterfly skin due to the extreme fragility of this tissue in ...
The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.
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Pharmaceutical Technology on MSNMeiraGTx and Hologen launch AI-backed gene therapy ventureThe venture aims to advance MeiraGTx’s Parkinson’s gene therapy through Phase III trials with up to $430m in funding from ...
10don MSN
Together, the manufacturer and the nonprofit are developing a gene therapy treatment that could change the lives of ...
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
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