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 ...
Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
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