The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
August 16, 2010 by CureDuchenne
Filed under Research Articles
“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 control information, to provide monetary or other incentives, and to communicate directly with biopharmaceutical companies and clinical investigators.”
To continue reading, please visit ClinicalTrialsFeeds.org.
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FAQs RE: ACE-031 on the Acceleron website
August 13, 2010 by CureDuchenne
Filed under Research Articles
The following is a link to FAQ’s on Acceleron Pharma’s website regarding ACE-031. To learn more click in this link:
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Details Regarding Study DMD114117 for Duchenne Muscular Dystrophy
August 13, 2010 by CureDuchenne
Filed under Research Articles
Details regarding several of the investigator sites for the following study have been posted to www.orpha.net (the portal for rare diseases and orphan drugs) today:
Study DMD114117: A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy
Additional study details, including inclusion/exclusion criteria and study endpoints are posted on www.clinicaltrials.gov. Investigator site details for this study, taking place in Europe, Turkey and Australia, will also appear on www.clinicaltrials.gov in the near future.
Sincerely,
John E. Kraus, MD, PhD Padraig Wright, MD, PhD
