The goal of this observational study is to characterize DMD at various stages of progression using the same measures used in ongoing clinical studies, such as the "six minute walk test." No medication is being tested in this study.
"The enrollment of the 100th patient
for the Natural History Study reinforces our commitment to
developing innovative treatment options for DMD," said Dr.
Patients in the study are assessed every six months for a period of three years to measure their muscle strength and function, in addition to how the disease affects their quality of life as the condition evolves over time. Investigators are observing the patients as they perform various physical tests, and assess their quality of life through survey questions. Furthermore, certain biomarkers are measured through blood and urine samples to investigate a possible correlation to disease progression.
Prosensa and GlaxoSmithKline (GSK), who are
both funding the study, expect to enroll up to 250 DMD
patients into the study with any type of mutation in the DMD gene
between the ages of three and 18. The study is being
conducted in 16 hospitals across 10 countries with 14 sites already
open in the U.S. and
Notes to editors
Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington's disease.
Prosensa's current portfolio includes six compounds for the
treatment of DMD, all of which have received orphan drug status in
Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 1 in 3,500 boys. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12. Few patients survive the age of 30.
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Prosensa Holding N.V. Celia Economides, Director IR & Corporate CommunicationsPhone: +1 917 941 9059 Email: firstname.lastname@example.org
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