Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...
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First Brain-Delivered Gene Therapy Approved for AADC DeficiencyThe treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...
On Thursday, Bluebird Bio Inc. (NASDAQ:BLUE) reported a third-quarter EPS loss of 31 cents, compared to a loss of 80 cents a ...
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
The Mass General Brigham Gene and Cell Therapy Institute, a hub of innovation dedicated to accelerating groundbreaking research, conducting clinical trials and developing FDA-approved treatments, ...
Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
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