gene therapy, Kebilidi

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 voucher for PTC.
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company. It is the first gene therapy directly administered to the brain and has received a broad label, allowing it to treat a wide ...
PTC Therapeutics' AADC Deficiency Gene Therapy Kebilidi Gains FDA Accelerated Approval
This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries,
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA's accelerated approval is based on the saf
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 more here.
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 and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.
US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
The U.S. Food and Drug Administration approved PTC Therapeutics' gene therapy to treat a potential fatal enzyme deficiency disorder, the company said on Wednesday, sending its shares up about 2% in aftermarket trade.
PTC Therapeutics’ AADC deficiency gene therapy granted FDA approval
PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever
Harvard Medical School2h
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 ...
Science Daily14h
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.
STAT25d
Perplexing results from Duchenne muscular dystrophy trial raise questions about gene therapies
Confounding data from a Pfizer clinical trial has rattled the field of gene therapy for Duchenne muscular dystrophy, raising more questions about the regulatory standard used to approve a ...