Ptc Therapeutics

This study tested a drug called PTC518 to see if it is safe and can reduce the levels of the harmful huntingtin protein that causes Huntington's disease. It involved 159 adults with early-stage Huntington's disease who received either the drug or a placebo for up to 12 months. Th…

This study aimed to check the long-term safety of a drug called ataluren in people with Duchenne muscular dystrophy caused by a specific 'nonsense' genetic mutation. It involved 270 participants who had already taken ataluren in a previous trial, or were siblings of those partici…

This study monitored the safety and real-world effectiveness of the drug Translarna (ataluren) for people with Duchenne Muscular Dystrophy. It followed 316 patients across Europe for at least five years as they received the drug as part of their normal medical care. The goal was …

This study monitored the safety of vatiquinone in 101 patients with inherited mitochondrial diseases who had previously taken the drug in earlier trials. It tracked side effects and adverse events to understand the long-term safety profile. The goal was to gather safety data unti…

This study tested whether a drug called ataluren could slow the progression of muscle weakness in boys with a specific genetic form of Duchenne muscular dystrophy. For 72 weeks, 360 participants received either ataluren or a placebo, followed by another 72 weeks where everyone re…