Lung Function in Ataluren-Treated, Nonambulatory Patients with Nonsense Mutation Duchenne Muscular Dystrophy from a Long-Term Extension Trial

A mini‐review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy

“…This recommendation is in line with the approval of the Committee for Medicinal Products for Human Use of the European Medicines Agency in May 2018 to expand the indications for ataluren to include ambulatory children aged two to five years with nmDMD. Moreover, based on the available data, which suggests that ataluren would be expected to have a non‐trivial, positive benefit on upper extremity and pulmonary functioning in patients with advanced disease , we propose that treatment with ataluren should continue until the patient has reached one of the following clinical milestones. These are a forced vital capacity below 30%, and, or, grade six on the Brooke upper extremity scale, for example if they cannot raise their hands to their mouth and their hands have no useful function.…”