Rare Disease Report

BioMarin's Gift to the Duchenne Community - Data

JANUARY 10, 2017
Andrew Black and James Radke
Last year, many in the Duchenne community were disappointed when BioMarin decided to discontinue their Duchenne program. That was the bad news. The good news is that the company is letting some of the data they collected be used to better understand Duchenne muscular dystrophy.
Toda it was announced that BioMarin has transferred their Duchenne natural history database to the non-profit group, CureDuchenne.
Specifically, CureDuchenne receives data on 270 DMD patients, ages 3-18-years-old disease progression experiences over a 3-year timetable. Results from the first year of the study are currently being prepared for publication.
The PRO-DMD natural history study, originally sponsored by Prosensa was continued by BioMarin when the latter bought the rights to Prosensa’s exon skipping drugs.
Debra Miller, CEO and founder of CureDuchenne said, “We are grateful to BioMarin for sharing their natural history database for the good of the entire Duchenne community.”
Ms Miller added, “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.”
Prof. Nathalie Goemans, a pediatric neurologist from the University Hospital of Leuven, Belgium and principal investigator of the PRO-DMD natural history study said, “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."
This information collected by BioMarin now given to CureDuchenne will hopefully lead to potential new clinical trials for DMD treatment and hopefully a step closer to a cure.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.
There are an estimated 35,000 patients with Duchenne muscular dystrophy in the United States and Europe but the population has many subsets based on mutations of the dystrophin gene.

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