All News #Library
Rare Diseases
PTC Withdraws US Approval Filing For Duchenne Drug Translarna
13 Feb 2026 //
FIERCE PHARMA
PTC Therapeutics Provides Regulatory Update On Translarna
12 Feb 2026 //
PR NEWSWIRE
PTC Therapeutics Gets Japan Approval For Sephience In (PKU)
22 Dec 2025 //
PR NEWSWIRE BIO
RareMed Announces Expansion of Relationship with PTC Therapeutics
02 Sep 2025 //
PR NEWSWIRE
PTC Therapeutics Receives CRL for Vatiquinone NDA
20 Aug 2025 //
PRESS RELEASE
Orsini Chosen as PTC Therapeutics` Sephience Pharmacy Partner
30 Jul 2025 //
PR NEWSWIRE
US FDA approves PTC Therapeutics` Metabolic Disorder Drug
29 Jul 2025 //
REUTERS
Sephience Gets EU OK for PKU Drug Sepiapterin
24 Jun 2025 //
PR NEWSWIRE
Sephience (Sepiapterin) Approved by European Commission for PKU
23 Jun 2025 //
PR NEWSWIRE
PTC Therapeutics Gets Positive CHMP Opinion for Sephience for PKU
25 Apr 2025 //
PR NEWSWIRE
FDA Priority Review To PTC’s Vatiquinone For Friedreich’s Ataxia
19 Feb 2025 //
PR NEWSWIRE
FDA approves PTC Therapeutics` gene therapy for ultra-rare disorder
14 Nov 2024 //
REUTERS
Technical Analysis of PTC Therapeutics
29 Apr 2024 //
TRADINGVIEW
PTC plots path to rare disease approval despite phase 3 fail
25 May 2023 //
FIERCE BIOTECH
PTC Therapeutics: A Gem In The Rare Diseases Treatment Market
10 May 2023 //
SEEKING ALPHA
Stuart Peltz, Ph.D., Rare Disease Pioneer, to Retire as CEO of PTC Therapeutics
24 Mar 2023 //
PR NEWSWIRE
NICE backs PTC Therapeutics` gene therapy for ultra-rare disease
24 Mar 2023 //
FIERCE PHARMA
NICE signs off on rare disease therapies from Alnylam, PTC
20 Jan 2023 //
FIERCE PHARMA
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
28 May 2020 //
HEMOPHILIANEWSTODAY
PTC Therapeutics Announces First-Ever Winners of Its PRIORITY Program
20 May 2019 //
PR NEWSWIRE
Idorsia sheds surplus DMD therapy to Santhera
21 Nov 2018 //
FIERCE BIOTECH
PTC Therapeutics to Acquire Agilis Biotherapeutics
20 Jul 2018 //
PR NEWSWIRE
PTC Therapeutics Announces R&D Day to Review Multiple Orphan Disorder Programs
09 Apr 2018 //
PRESS RELEASE

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