RG3039 is the first clinical-stage drug candidate to target the core genetic deficit in SMA in order to treat the biochemical deficits caused by decreased levels of the survival motor neuron (SMN) protein. This key protein is necessary for normal neuromuscular function but is insufficiently produced in SMA patients. RG3039 is an orally bioavailable small molecule inhibitor of an RNA processing enzyme called DcpS. RG3039 has been shown to increase production of the SMN protein in cells derived from patients. In addition, RG3039 has been shown to improve motor neuron pathology, mobility and lifespan in animal models of SMA.Top-line results from this Phase 1 study of RG3039 are scheduled to be presented as part of a special neuroscience program at the 64 th Annual Meeting of the American Academy of Neurology (AAN). The AAN meeting is being held April 21-28, 2012 at the New Orleans Ernest N. Morial Convention Center. James P. Van Meerbeke, Research Assistant from the lab of Charlotte J. Sumner, M.D., Associate Professor of Neurology and Neuroscience, Johns Hopkins University School of Medicine, will present the abstract titled “ The Therapeutics Effects of RG3039 in Severe Spinal Muscular Atrophy – Mice and Normal Human Volunteers,” during “The Future of Neuroscience Conference: Neurologists and Neuroscientists Defining the Next Generation of CNS Therapies,” taking place on April 27.
Repligen Reports Positive Results From Phase 1 Clinical Trial Of RG3039 For Spinal Muscular Atrophy
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