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747,540 Results for "ptc therapeutics inc".
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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
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
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 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
Business
PTC Therapeutics Provides Corporate Update and Reports First Quarter 2024 Financial Results
PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2024.
April 25, 2024
·
14 min read
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