PTC Therapeutics

Related records

Trials (76)

• A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
• A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
• A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
• A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
• A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
• A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
• A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
• A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
• A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
• A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
• A Study of CNSA-001 in Women With Diabetic Gastroparesis
• A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
• A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
• A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
• A Study of PTC923 in Participants With Phenylketonuria
• A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
• A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
• A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
• A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
• A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia