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 Wednesday, sending its shares up about 2% in aftermarket trade.
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.
First Brain-Delivered Gene Therapy Approved for AADC Deficiency
The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have decreased AADC activity that causes severe disability, including gross motor function deficits,
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 vector-based gene therapy indicated for the treatment of adult and
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 (AADC) deficiency.
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.
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
William Blair analyst Sami Corwin has maintained their bullish stance on PTCT stock, giving a Buy rating on November 7. Sami Corwin has given
PTC Therapeutics wins FDA nod for gene therapy
PTC Therapeutics (NASDAQ:PTCT) has received FDA approval for its gene replacement therapy Kebilidi for the treatment of children and adults with AADC deficiency, a rare genetic disorder that impairs the ability to generate dopamine,
Alternative experimental gene therapy restores hearing, boosts vision in tests
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
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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.
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Muscular dystrophy gene therapy uses cells to stitch together mRNA strands in mice
Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...
Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
STAT1d
Neurogene and the race to the bottom of gene therapy
At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.
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Patent Vs. Trade Secret Considerations For Cell And Gene Therapies
Understanding what to patent versus what to keep as a trade secret is an increasingly important and challenging decision ...
Gene therapy protects against motor neuron disease in rats
University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...
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 ...