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716,063 Results for "ptc therapeutics inc".
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Huntington’s disease
PTC Nosedives Despite Claiming Phase II Win for Huntington’s Hopeful
The biotech’s Huntingtin-targeting molecule lowered blood levels of the protein and elicited functional improvements in earlier-stage patients, but results were not as robust in other biomarkers or with patients at later stages of the disease.
May 5, 2025
·
2 min read
·
Dan Samorodnitsky
Pharm Country
PTC Therapeutics to Participate at Upcoming May 2024 Investor Conference
PTC Therapeutics, Inc. announced that the company will present a company overview at the following conference.
May 29, 2024
·
1 min read
Pharm Country
PTC Therapeutics Announces Validation of Sepiapterin European MAA
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA.
May 28, 2024
·
6 min read
Huntington’s disease
Novartis Pays Up to $2.9B in Huntington’s Deal With PTC
Monday’s agreement comes days after PTC discontinued the development of another asset, utreloxastat, due to disappointing Phase II data in amyotrophic lateral sclerosis.
December 2, 2024
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2 min read
·
Tristan Manalac
Pharm Country
PTC Therapeutics to Participate at Upcoming May 2024 Investor Conferences
PTC Therapeutics, Inc. announced that the company will present a company overview at the following conferences.
May 2, 2024
·
1 min read
Approvals
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene therapy to be delivered directly to the brain.
November 14, 2024
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2 min read
·
Tristan Manalac
Neurodegenerative disease
PTC Scraps ALS Asset Utreloxastat After Mid-Stage Fail, Analysts Look to PKU Opportunity
Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect PTC, instead postulating that the upcoming FDA decision on its phenylketonuria candidate sepiapterin will be a stronger driver of the biotech’s stock.
November 27, 2024
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2 min read
·
Tristan Manalac
Pharm Country
PTC Therapeutics Announces FDA Acceptance and Priority Review of the BLA for Upstaza™
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of AADC deficiency.
May 14, 2024
·
6 min read
Policy
PTC Therapeutics Provides Key Regulatory Updates
PTC Therapeutics, Inc. announced the submission of a BLA to the U.S. FDA for Upstaza™, a gene therapy for the treatment of aromatic L-amino acid decarboxylase deficiency.
March 19, 2024
·
8 min read
Pharm Country
PTC Therapeutics to Participate at Upcoming March 2024 Investor Conferences
PTC Therapeutics, Inc. announced that the company will present a company overview at the following conferences.
February 27, 2024
·
1 min read
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