Daily4h
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 ...
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., ...
Monthly Prescribing Reference22h
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 ...
MM&M6h
Rx Rundown: Roche, Metsera, Intercept Pharmaceuticals and more
AstraZeneca and Amgen announced that a Phase 3 trial of Tezspire (tezepelumab) showed a statistically significant and ...
After raising nearly half a billion, ABL Space pivots from launch vehicles to missiles
Significant changes are coming to ABL Space Systems. The leader of the seven year old startup took to social media last night ...
BioPharma Dive21h
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 ...
Roundtable with Shaw Laureates
Breakthroughs and Impacts", presented jointly by The Shaw Prize Foundation and the Hong Kong Science Museum, and organised by ...