Working for the biotech company didn’t help this women get access for her son, even though she was one of their biggest fans and promoters, now she’s on the other side, suing as she stated her son would be given access to the trial. She is appealing for a compassionate use provision. The attending pediatrician is behind her as well and states it could possibly save her son’s life.
When her son was still able to walk the company officials said not to enroll him in a preliminary 28-day study to determine if the drug had any effect at all, and a later enrollment would be an option. Who gets access and who does not? BD
She also says she worked on behalf of PTC Therapeutics, the biotechnology company that is developing what she thinks is the most promising treatment for a genetic mutation aimed at about 15 percent of the boys with Duchenne. Many of the boys and young men who have the disease — it is nearly always a male condition — do not live past their 20s.
Because Jacob is no longer walking, he is not eligible for the clinical trial under way, which involves about 165 patients. And the company says it will not provide the experimental compound, PTC124, to any boy who is not involved in one of the drug’s studies.
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