Austin, 13, Max, 10. Two little brothers with red hair and a face full of freckles seemed united by the same fate, both affected by a rare progressive genetic disease called "Duchenne muscular dystrophy". Today, however, Max is recovering strength, flexibility, the ability to walk, to open jars, to play: normal activities, but which are almost impossible for the victims of the disease. Austin on the other hand doesn't even move from the bed and the wheelchair anymore. Raising a glass of water is out of the question.
THE DRUG AND THE REPLY – For 52 weeks Max has been receiving infusions of the experimental drug based on "eteplirsen" from the pharmaceutical company "Sarepta Therapeutics", and the therapy - not yet approved - is working wonders in his case. There are 12 other kids like him under test. Brother Austin, on the other hand, was denied the same drug because he was no longer able to walk and therefore did not meet the criteria for experimentation. Despite the prayers, tears and requests of her mother, Jan Mcnary, there was nothing to be done: "The managers of the pharmaceutical company have written to me only to understand my case," the woman told the American media. In an email to the family, Sarepta Therapeutics wrote: "Giving this medicine not only to Austin but to all the children who could benefit from it remains our priority. But there are complex tax, legal, political and production issues that need to be resolved. Meanwhile, according to the family, Austin's condition is deteriorating to the point that in the coming months he will need a machine to help him breathe in his sleep.
August 22, 2012 | 11:53am The Corriere della Sera Salute
