Ambulatory Duchenne patients who are 5 years and older with a nonsense mutation can now access a treatment that targets the underlying cause –
SOUTH PLAINFIELD, N.J., April 29, 2019 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna™ (ataluren) has been granted marketing approval from the Brazilian National Health Surveillance Agency (ANVISA) under rare diseases procedure, for the treatment of ambulatory children five years and older with Duchenne muscular dystrophy caused by a nonsense mutation. Patients now can have access to a treatment that targets the underlying cause.
«The regulatory approval from the Brazilian authorities will accelerate access to Translarna for the many patients who have been waiting for treatment,» stated Eric Pauwels, Senior Vice President and General Manager of the Americas of PTC Therapeutics, Inc. «We are committed to working quickly to make Translarna available to all patients in Brazil who may benefit.»
#DMD #Duchenne #muscular dystrophy