Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy

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Dr. Chris Lorson and his colleagues have published a paper, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy,” in the journal Molecular Therapy. The University of Missouri-based team is investigating ways to target SMN2, the SMA “backup gene.” Because of a genetic mutation in the SMN1 gene, individuals with […]

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AveXis Reports Second Quarter 2016 Interim Data from Ongoing Phase 1 Trial of AVXS-101 (Gene Therapy)

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AveXis recently released new data from the Phase 1 clinical trial of AVXS-101, a gene therapy program to treat SMA. The interim data through July 1 showed no “events.” The data also showed continued motor function improvement. AveXis has requested a Type B Meeting with the FDA to discuss SMA Type 1 clinical development pathway.

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2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA

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The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research. The meeting also furthers research by building productive collaborations—including cross-disciplinary

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Novartis Releases Community Update on the Study of LMI070

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Novartis recently provided a community update on clinical trials for LMI070. This information is effective as of July 25 2016: “In May, we shared with you news of the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). This decision was made because results

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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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