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Connect with Families of SMA: 
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| 2011 Research News |
| February 2, 2012 |
The Lancet Neurology Article on NeuroNEXT: accelerating drug development in neurology
In February, 2012, the US National Institute for Neurological Disorders and Stroke (NINDS) is due to announce the first study to be supported by the National Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT). he first NeuroNEXT study will be an investigation of potential biomarkers to assess treatments for spinal muscular atrophy (SMA).
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| January 18, 2012 |
Paper Showing Direct CNS Delivery of Gene Therapy Provides Enhanced Protection in a Severe Mouse Model of SMA.
Gene replacement strategies have shown great promise in animal models of SMA. In this study, scAAV9-SMN was used to compare two different routes of viral delivery in a severe SMA mouse model. This was accomplished by injecting scAAV9-SMN vector intravenously (IV) -often referred to as systemic delifvery, or intracerebroventricularly (ICV) - direct CNS delivery, into SMA mice.
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| October 21, 2011 |
Vertex Success in CF Drug Development Leads the Way for Other Orphan Diseases: NDA filed with the FDA
This week Vertex Pharmaceuticals announced filing a New Drug Application with the FDA for a disease modulating Cystic Fibrosis Drug. Importantly, this work provides proof-of principle that small molecule drug development is possible for other orphan, genetic diseases, including for Spinal Muscular Atrophy.
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| October 13, 2011 |
Charlotte Sumner and James Van Meerbeke at Johns Hopkins University Publish Article Reviewing the Spinal Muscular Atrophy Drug Pipeline.
Dr. Sumner is a member of the FSMA Translational Advisory Committee and a 2007/2008 grant recipient from FSMA.
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| September 28, 2011 |
Article In Regenerative Medicine Explains Potential of Stem Cell Therapies for ALS and SMA.
Authors Eva Feldman, Nicholas Boulis, and colleagues present supporting evidence for the use of stem cell technology in the treatment of amyotrophic lateral sclerosis and spinal muscular atrophy in the Journal Regenerative Medicine. They also discuss the first-in-human Phase I trial currently underway examining the safety and feasibility of intraspinal stem cell injections in ALS patients as a foundation for translating stem cell therapies for various neurological diseases. In September 2011, NeuralStem Inc. released interim data on this ALS trial.
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| September 13, 2011 |
New Ipierian CEO Renews Commitment to Spinal Muscular Atrophy Drug Discovery
Please click here to read more about the article in San Francisco Business Journal.
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| July 26, 2011 |
New insights into a possible therapeutic approach to treating SMA
The current issue of the Journal of Clinical Investigation includes two publications addressing SMN enhancing approaches for SMA.
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| July 26, 2011 |
Summary of Dr. Jill Jarecki's Talk at the Annual SMA Conference
Dr. Jarecki, FSMA Research Director, gave a talk on FSMA Research Activities and Investments. Please click here to read a summary of her talk.
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| July 26, 2011 |
Summary of Dr. Douglas Kerr's Talk at the Annual SMA Conference
Dr. Kerr from Biogen Idec gave a talk on The Drug Development Process. Please click here to read a summary of his talk.
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| July 26, 2011 |
Summary of Dr. Charlotte Sunmer's Talk at the Annual SMA Conference
Dr. Sumner from Johns Hopkins University gave a talk on New Basic Research Developments in SMA. Please click here to read a summary of her talk.
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