Rare diseases
Investigational molecules
Locations
500 Warren Corporate Center Drive, 07059, United States
PTC Therapeutics Inc. supports clinical research in amyotrophic lateral sclerosis and Huntington’s disease, with attention to disease progression, neurological function, and biomarker changes linked to central nervous system involvement.
These studies reflect a focus on conditions with progressive motor and cognitive decline.
PTC Therapeutics Inc. runs clinical trials in conditions such as Phenylketonuria, Friedreich's ataxia, Neoplasm, Autoimmune disorder, among others.
Clinical trials run by this sponsor – those enrolling participants first.
Study of Long-Term Neurocognitive Outcomes in Children with Phenylketonuria Treated with Sepiapterin
FranceLong-term safety and effectiveness study of vatiquinone (oral capsules) in patients with Friedreich ataxia who have previously received this treatment
FrancePhase 1 study of oral and intravenous unesbulin to assess absorption, metabolism and excretion in patients with solid tumors
HungarySafety and Pharmacokinetics of Oral PTC844 in Healthy Participants for Autoimmune Disorder Treatment
The NetherlandsLong-Term Safety Study of ST-920 Gene Therapy for Patients with Fabry Disease
GermanyStudy on the Long-Term Safety of PTC923 (Sepiapterin) for Patients with Phenylketonuria
CzechiaStudy of utreloxastat (PTC857) in adults with Amyotrophic Lateral Sclerosis (ALS) to evaluate its effectiveness in slowing disease progression
BelgiumStudy Comparing Sepiapterin and Sapropterin for Treating Phenylketonuria in Patients Aged 2 Years and Older
CzechiaStudy on the Safety and Effects of PTC518 for Patients with Huntington's Disease
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