Research

2016 SMA Researcher Meeting Summary: Clinical Research Studies

August 23, 2016
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We will be posting a series of summaries from our 2016 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]

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

August 17, 2016
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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 […]

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

August 12, 2016
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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 […]

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2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA

August 11, 2016
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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. […]

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

August 4, 2016
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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 […]

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

August 1, 2016
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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 […]

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Cure SMA Announces $2.5 Million in New Planned Research Funding

July 25, 2016
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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 […]

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

July 14, 2016
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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” […]

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Adelphi Values’ RO7350A Interview Study

July 7, 2016
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A note from Cure SMA: We’re passing on information about this new interview study to the families in our community. It provides an opportunity to […]

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

July 6, 2016
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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 […]

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