PTC Therapeutics announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna will be made available to these siblings in advance of commercial availability in their regions, after a treating physician and the patient or guardian make a clinical decision to use Translarna, and consistent with any other applicable regulatory requirements.

"For families with more than one child suffering from nonsense mutation Duchenne muscular dystrophy, having only one child enrolled in our clinical trials with access to Translarna, can cause great distress for parents," says Stuart W. Peltz, chief executive officer, PTC Therapeutics, Inc. "Knowing this, we worked to find a solution to ensure those siblings who were not eligible to participate in the trials can still have similar access. We are eager to roll out this programme and are committed to providing access to Translarna for all who may benefit."

Kate Bushby, Professor of Neuromuscular Genetics at Newcastle University says, "Though Duchenne muscular dystrophy is a rare disorder, the nature of an inheritable genetic disease means that there is an increased likelihood that siblings will be born with the same mutation. Translarna is the first treatment option available to treat the underlying cause of nonsense mutation DMD. It is exciting to see PTC's commitment to the patients contributing towards our understanding of the natural history of Duchenne and developing Translarna as a treatment for nmDMD."

"Families of children with Duchenne have wanted companies to be responsive in the case of siblings," added Pat Furlong, Founding President of Parent Project Muscular Dystrophy. "With this programme, PTC, once again, demonstrates itself as a champion for the Duchenne community."

Translarna, 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 is licenced in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States. The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.