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Congratulations to Dr. Lee Rubin for His Recent Publication in Nature Chemical Biology
…compartments of intact cells. As a result of this screen, Dr. Rubin’s group discovered several classes of compounds that elevate SMN protein levels in SMA patient fibroblasts and motor neurons…
Read MoreSMA Foundation comments to FDA on the importance of combination therapy for rare diseases like SMA
…rare disease community. We hope to see the FDA take steps under FDASIA to encourage development of combination therapies. For the Foundation’s full comment, please see the following link: http://www.regulations.gov/#!documentDetail;D=FDA-2014-N-1698-0021…
Read MoreLeading Medical Institutions Establish a Clinical Research Network to Study SMA
…diseases such as SMA,” commented Dr. De Vivo, Sidney Carter Professor of Neurology at Columbia University Medical Center and Director of the SMA Clinic. “With no current treatments available for…
Read MoreSMA Foundation Funds Over $15 Million to Develop Treatments for SMA
…leading to muscle weakness, respiratory complications and premature death. Having been identified by researchers as the neurological disease closest to a treatment, SMA has been selected by the National Institute…
Read MoreSMA Foundation Makes the Case for Research in Rare Disease
…and CombinatoRx, with the goal of new compounds entering clinical testing as early as 2006–07. “This really is a win-win,” said Alexis Borisy, president and CEO of CombinatoRx, a young…
Read MoreNew Motor Neuron Center Will Tackle Neurodegenerative Diseases
…when Columbia’s Motor Neuron Center was launched. The gathering of about 40 leading researchers and clinicians from different disciplines at CUMC and Morningside, committed to sharing knowledge, insights, and current…
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