CureDuchenne announced today that BioMarin Pharmaceutical Inc. has transferred its Duchenne natural history database to CureDuchenne, the venture philanthropy not-for-profit organization that is leading Duchenne research. CureDuchenne will house and maintain BioMarin’s natural history database, and will facilitate sharing of currently warehoused data and collaborate with other institutions as well, to maximize our knowledge of the natural progression of this devastating muscle disease.
BioMarin conducted a natural history study designed to prospectively evaluate the progression of patients with Duchenne muscular dystrophy. This observational study of nearly 270 pediatric patients (between three and 18 years of age) was designed to characterize the progression of Duchenne over a three-year period to provide information that may help in the development of therapeutic clinical trials. The results of the 12-month interim analysis of 77 ambulatory patients further describes the relationship between baseline characteristics, including age and six-minute walking distance and the trajectory of disease progression. These findings are consistent with that reported in other natural history studies.
“We are grateful to BioMarin for sharing their natural history database for the good of the entire Duchenne community,” said Debra Miller, CEO and founder of CureDuchenne. “Understanding the progression of the disease is critical in the design and analysis of clinical trials that will lead to potential treatments for Duchenne. The data sharing is invaluable in the effort to find a cure for this devastating disease.”
“BioMarin is pleased to provide our natural history database to CureDuchenne,” said Hank Fuchs, M.D., President, Worldwide Research and Development, BioMarin. “CureDuchenne is a leading advocacy group for the Duchenne community and they continue to work with leading investigators who have worked with the data. Access to the data will allow data sharing among academics and the industry for those working on treatments for Duchenne.”
The PRO-DMD natural history study, originally sponsored by Prosensa, and subsequently continued by BioMarin, has collected three years of longitudinal natural history data on children and adults with DMD in a multicenter international collaboration. Results from the first year of the study are currently being prepared for publication. Analysis and preparation for publication of two- and three-year data will follow.
Prof. Nathalie Goemans, a pediatric neurologist from the University Hospital of Leuven, Belgium and principal investigator of the PRO-DMD natural history study stated: “We are pleased that CureDuchenne has obtained the database from BioMarin and has pledged to collaborate with the PRO-DMD investigators. The patients and families participating in this study have given significant time and effort to help us understand the progression and burden of DMD. Our transatlantic partnership between investigators at institutions throughout the E.U., Canada and the United States, including Prof. Craig McDonald from UC Davis and Prof. Eugenio Mercuri from the Catholic University in Rome, represents a significant effort for all involved.”
CureDuchenne was founded in 2003 with a focus on saving the lives of those with Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne also is the innovator bringing physical therapy and standard of care to local communities around the country throughCureDuchenne Cares. For more information, please visit CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.