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 ...
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 ...
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
The San Diego biotech made an unsolicited bid to buy Evotec for 2 billion euros. Elsewhere, Bluebird recorded its first ...
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...
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