FDA, genetic disorder and PTC Therapeutics

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,
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 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,
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.,
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
Sami Corwin has given his Buy rating due to a combination of factors that favor PTC Therapeutics’ market position. The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company.
PTC gets FDA okay for first brain-delivered gene therapy
Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during a surgical procedure, and replaces the human dopa decarboxylase (DDC) gene that is mutated in the disease, allowing dopamine synthesis to take place.
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
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 deficiency treatment.
FDA nod for AADC deficiency gene therapy from PTC
The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ gene therapy for the treatment of AADC (aromatic L-amino acid decarboxylase) deficiency, making it the first-ever gene therapy approved in the USA that is directly administered to the brain.
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’ 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
PTC Therapeutics price target raised to $45 from $43 at Barclays
Barclays raised the firm’s price target on PTC Therapeutics (PTCT) to $45 from $43 and keeps an Equal Weight rating on the shares. After ...
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FDA approves first gene therapy delivered directly to the brain
The therapy, developed by PTC Therapeutics, is designed for patients across the severity of spectrum of AADC deficiency.
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PTC Therapeutics, Inc.: PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
Klein, M.D., Chief Executive Officer, PTC Therapeutics. "I am proud of our team's unwavering commitment to achieve this important regulatory milestone. We look forward to bringing this ...
ClearPoint Neuro Announces FDA De Novo Marketing Authorization of SmartFlow Cannula for Direct Delivery of Gene Therapy to the Brain
Modifying Treatment for AADC Deficiency in the United States SOLANA BEACH, CA / ACCESSWIRE / November 13, 2024 / ...
Balancing Growth Prospects and Regulatory Challenges: A Hold Rating on PTC Therapeutics
Joseph Schwartz, an analyst from Leerink Partners, reiterated the Hold rating on PTC Therapeutics (PTCT – Research Report). The ...
PTC Therapeutics, Inc. (NASDAQ:PTCT) Q3 2024 Earnings Call Transcript
Q3 2024 Earnings Call Transcript November 7, 2024 PTC Therapeutics, Inc. beats earnings expectations. Reported EPS is $-1.39, ...
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After Decades of Failure, First Disease-Modifying Huntington’s Treatment on the Horizon
The past four years have brought disappointment for the Huntington’s community, but optimism is growing as companies ...
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Dianthus Therapeutics’ Strategic Advancements and Accelerated Drug Development Drive ‘Buy’ Rating
Beatty covers the Healthcare sector, focusing on stocks such as PTC Therapeutics, Longboard Pharmaceuticals, and Alkermes. According to TipRanks, Beatty has an average return of 14.7% and a 53.75% ...