Sebastien Beauzile, 21, became the first person in New York state to be made symptom-free of sickle cell disease after ...
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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 ...
Cellular therapy, according to the Dana-Farber Cancer Institute, is designed to improve the immune system’s ability to fight ...
The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...
Mía had dystrophic epidermolysis bullosa, better known as butterfly skin due to the extreme fragility of this tissue in ...
Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
With MeiraGTx eyeing up how to take the drug through phase 3 and onto commercialization, Hologen has stepped in. The ...
X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in ...
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...
Aaron Scott, of Fayetteville, is the first in North Carolina to receive a new FDA-approved therapy which uses the patient's own cells to relieve the pain of sickle cell disease.
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