AveXis Receives FDA Breakthrough Therapy Designation for AVXS-101 (Gene Therapy) for SMA

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AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. The Breakthrough […]

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Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports

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Dr. Rashmi Kothary and his team have recently published a paper, “Differential induction of muscle atrophy pathways in two mouse models of spinal muscular atrophy” in the journal Scientific Reports. This research was funded in part by grant from Cure SMA. Individuals with SMA do not produce survival motor neuron (SMN) protein at high enough

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Cure SMA Awards $50,000 Drug Discovery Grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences

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Cure SMA has awarded a $50,000 drug discovery grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences. The award is for the project, “Slowing SMN degradation to treat SMA.” Individuals with SMA don’t produce survival motor neuron (SMN) protein at high enough levels due to a mutation in the survival motor

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Cure SMA Releases 2016 Updates from Drug Programs in Clinical Trials

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At the Annual SMA Conference, representatives from the six programs currently in clinical trials gave an update on their programs. Of these six programs, four— AVXS-101 (gene therapy), nusinersen, RG7800/RG7916, and LMI070—treat the underlying genetics of SMA. Gene therapy aims to replace the mutated in the SMN1 gene that causes SMA, and the other three

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Cure SMA Posts Keynote Presentations on the NDA Process

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The ultimate goal in drug development is to get treatments approved for as many people as possible, as quickly as possible. Because these issues are complex, we want our community to be knowledgeable about the burden of proof required to support an FDA-approved treatment, and how that approval process works. As we get closer to

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Cure SMA Awards $304,000 Drug Discovery Grant to Livio Pellizzoni, PhD, at Columbia University

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Cure SMA has awarded a $304,000 drug discovery grant to Livio Pellizzoni, PhD, at Columbia University. The award is for the project, “Pharmacological Inhibition of p38αMAPK as a Candidate Therapeutic Approach for SMA.” About the Project Dr. Pellizzoni and his colleagues at Northwestern University have identified a novel cellular pathway, called p38αMAPK, that is altered

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