BioPharma Dive1d
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 ...
FDA Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency
The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the ...
The American Journal of Managed Care1d
FDA Approves Gene Therapy to Treat AADC Deficiency
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
Harvard Medical School1d
Second Gene Therapy Shows Promise for Syndrome Involving Blindness, Deafness
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...
PTC Therapeutics wins FDA nod for gene therapy
PTC Therapeutics (PTCT) stock on watch as company receives FDA approval for gene therapy Kebilidi for AADC deficiency. Read ...
Science Daily1d
New discovery enables gene therapy for muscular dystrophies, other disorders
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult ...
Reuters2d
US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to treat a potential fatal enzyme deficiency disorder, the company said on ...
Yahoo Finance3d
PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the ...
dermatologyadvisor22h
Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa Under Review
The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.