Author name: Morgan Tuttle

Clinical Trial of CK-2127107 in Patients with Spinal Muscular Atrophy

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CK-2127107 is a novel fast skeletal muscle troponin activator being developed as a potential treatment for people living with SMA and certain other debilitating neuromuscular and non-neuromuscular conditions associated with muscle weakness and/or muscle fatigue. CK 2127107 is liquid form taken twice a day by mouth. This first clinical trial of this investigational drug in […]

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Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings

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Today, Biogen and Ionis Pharmaceuticals provided a statement regarding the decision to submit the first ever SMA regulatory filings for FDA approval. Dear Members of the SMA Community, Today marks an important day in our collective pursuit of a meaningful treatment for Spinal Muscular Atrophy (SMA). We are pleased to share the exciting news that

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Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA

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Biogen and Ionis have been testing nusinersen in clinical trials and today announced that they will close ENDEAR, the Phase 3 trial testing nusinersen in infants with SMA type 1. The trial met the primary endpoint pre-specified for the interim analysis of ENDEAR, the Phase 3 trial evaluating nusinersen in infantile-onset (consistent with Type 1)

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Cure SMA Announces $2.5 Million in New Planned Research Funding

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At the 2016 Annual SMA Conference, Cure SMA announced $2.5 million in new planned research funding over the next 12 months. This funding will be used strategically to help accelerate research and ensure we are developing treatments for all types, ages, and stages of SMA. Funding Priorities As the SMA research landscape has developed and

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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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