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FDA approves first-ever gene therapy to be directly administered to the brain
PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs ...
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Serious adverse event causes problems for Neurogene’s Rett gene therapy
A serious adverse event in the New York biotech’s phase 1/2 trial of its Rett syndrome gene therapy puts a question mark over ...
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Innovative COA tools to reduce steroid toxicity in autoimmune care
Collaboration to leverage Steritas’ STOX Suite in developing innovative treatments for autoimmune diseases.