Daily3h
FDA Approves First Gene Therapy for Treatment of AADC deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...
Roundtable with Shaw Laureates
Breakthroughs and Impacts", presented jointly by The Shaw Prize Foundation and the Hong Kong Science Museum, and organised by ...
Pharmabiz14h
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval
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., ...
BioPharma Dive20h
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
Monthly Prescribing Reference21h
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
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 ...
BioWorld23h
Healing a health divide (that’s long overdue)
At the BioFuture 2024 conference held in New York in November, Seema Kumar, the CEO of Cure, described women’s health as something that has been directed at the “bikini area.” That “bikini” bias ...