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PTC Therapeutics, Inc.

http://www.ptcbio.com/

Latest From PTC Therapeutics, Inc.

Upstaza: First Gene Therapy For Ultra-Rare Disorder AADC Deficiency Receives English Funding

The single-dose gene therapy for aromatic L-amino acid decarboxylase in children is to be made available on the National Health Service following a confidential commercial deal between NHS England and PTC Therapeutics.

Europe United Kingdom

MorphoSys Pins Hopes On Pelabresib For Near-Term Value After Tumultuous 2022

The German biotech has highlighted its myelofibrosis drug candidate, pelabresib, as a key value driver going forward after a year of hard knocks and lackluster Monjuvi sales that forced the firm to downsize its workforce and cease preclinical operations.

Cancer Sales & Earnings

Clene Confident In Phase III Plans For CNM-Au8 In ALS With Six-Month Data

The company announced detailed data for the drug in the HEALEY-ALS Platform Trial, where the cohort of the Phase II/III study testing it nevertheless failed to meet the primary endpoint.

Business Strategies Clinical Trials

Prilenia Plans To Continue With Pridopidine In ALS Despite Platform Study Miss

Pridopidine is the latest ALS drug candidate to miss the primary endpoint in the HEALEY-ALS platform study, but the S1R agonist did indicate potential benefits in speech and a key biomarker of the disease.

Clinical Trials Drug Approval Standards
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    • Antisense, Oligonucleotides
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