Share 'The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor'
[Reference: http://clinicaltrials.gov/ct2/show/NCT01182324?term=ataluren&rank=10 ]
The traditional process for the development of new potential therapeutics for rare disorders and the ensuing research on safety and efficacy is changing. This is due, in part, to the increased ability of families, parents, individuals with rare disorders, and advocacy organizations to access and con…
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