Ptc Therapeutics

This study tested an experimental drug called vatiquinone in 146 people with Friedreich ataxia, a rare disease that affects movement and coordination. Participants took the drug or a placebo for 72 weeks to see if it slowed worsening of symptoms, measured by a standard rating sca…

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

This study tested the safety of a drug called ataluren in 270 people with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). Participants took the drug three times daily at different doses. The main goal was to check for side effects and lab cha…

This study tested an experimental drug called PTC518 in 159 adults with Huntington's disease. The main goal was to check the drug's safety and how it affects a key protein linked to the disease. Researchers also looked at brain scans and movement scores to see if the drug might s…

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 followed 316 people with Duchenne muscular dystrophy who were already taking Translarna (ataluren) 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 informa…