For many of the thousands of orphan diseases, not only is there no effective treatment, but there is no existing FDA guidance on the regulatory requirements for obtaining approval for a new one. The Parent Project Muscular Dystrophy (PPMD) has taken the reins in their hands and submitted the first draft clinical trial guidance document ever submitted to the FDA by a parent advocacy group. The PPMD, which is comprised of parents of children with Duchenne muscular dystrophy (DMD), worked with a wide range of stakeholders, including academics from the U.S. and E.U., industry partners and DMD advocacy groups to draft the document outlining clinical strategies and potential surrogate endpoints for a clinical trial. Hopefully this will encourage other patients groups to follow suit – the ALSA has today announced such an initiative to develop guidance for ALS.
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