Ptc Therapeutics

This study looked at the safety of vatiquinone in 101 people with inherited mitochondrial disease who had already taken the drug in earlier studies. Participants continued treatment until the drug became available or the program ended. The main goal was to track any side effects,…

This study tested a drug called vatiquinone in 146 people with Friedreich ataxia, a rare nerve disease that affects movement and balance. The goal was to see if the drug could slow down the disease and improve daily function. Participants took the drug or a placebo for 72 weeks, …

This study tested a drug called ataluren in 360 boys (age 5 and older) with Duchenne muscular dystrophy caused by a specific genetic mistake called a nonsense mutation. The goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participa…

This study tested a new drug called PTC518 in 159 people with Huntington's disease. The main goal was to see if the drug is safe and if it can lower the level of a harmful protein (huntingtin) in the blood. Researchers also looked at brain scans and symptom changes over 12 months…

This study looked at the safety of a drug called ataluren in 270 people with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies or were siblings of those who did. The goal was to see how…

This study followed 316 people with Duchenne muscular dystrophy who were already taking the drug Translarna as part of their normal care. Researchers tracked side effects and how well the drug worked in real life, not in a controlled trial. The goal was to gather safety informati…