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288,209 Results for "ptc therapeutics".
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Huntington’s disease
Novartis, PTC push Huntington’s drug into Phase 3, mum on accelerated approval
Phase 2 data from PTC Therapeutics showed that the Novartis-partnered Huntington’s disease asset slowed progression by more than 50%. Analysts say the decision to initiate a last-stage trial reflects a lack of confidence in an accelerated FDA nod.
April 29, 2026
·
2 min read
·
Tristan Manalac
Press Releases
PTC Therapeutics Announces Health Canada Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
December 10, 2025
·
3 min read
Rare diseases
FDA Rejects PTC Therapeutics’ Friedreich’s Ataxia Drug
The small molecule, vatiquinone, had already flunked a Phase III trial, but the company pushed ahead with an approval bid anyway.
August 19, 2025
·
1 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
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
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
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
·
2 min read
·
Tristan Manalac
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
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
·
2 min read
·
Tristan Manalac
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