PTC Therapeutics, Inc.
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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.
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.
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.
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.
Company Information
- Industry
- Biotechnology
- Pharmaceuticals
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Biotechnology
- Antisense, Oligonucleotides
- Drug Discovery Tools
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Drug Discovery Tools
- Genomics-Proteomics
- Other Names / Subsidiaries
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- Agilis Biotherapeutics, Inc.
- Censa Pharmaceuticals Inc.
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