PTC Announces Expansion of Translarna™ Label to Include Ambulatory Patients as Young as 2 Years Old in Brazil

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[{"type":"text","content":"SOUTH PLAINFIELD, N.J., Oct. 25, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Brazilian National Health Surveillance Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved the expansion of the indication of Translarna™ (ataluren) to include ambulatory children aged 2 to 5 years old with nonsense mutation Duchenne muscular dystrophy (Duchenne).1 This broadens the use beyond the previous age range of 5 years and older. \n\n \n \n \n \n \n \n\n \n\"Expanding access to Translarna to children with nonsense mutation Duchenne in Brazil is important to us,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. \"We know that with Duchenne, early diagnosis and treatment gives children the best chance of preserving muscle function and delaying the progression of the disease.\"\nDuchenne is a severe progressive disease that leads to rapidly worsening muscle function with children often using a wheelchair by early adolescence and eventually requiring artificial ventilation to breathe.2,3 Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation. Clinical trials and real-world evidence have demonstrated Translarna's potential to slow disease progression and improve patient outcomes.1-3\nAbout Translarna (ataluren)Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna, the tradename of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States. \nAbout Duchenne Muscular Dystrophy (Duchenne)Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respira...

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