Trace Neuroscience, which launched this week with $101 million, benefited from last year’s approval of Qalsody. Its CEO spoke ...
The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.
Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...
Immunotherapy, which enhances the immune system's T cell response to eliminate cancer cells, has emerged as a key approach in ...
Why one trial participant's healthy birth after her CAR-T treatment for lupus is catching resesarchers' attention: ...
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
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