Research

2017 Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Disease Modifiers

August 24, 2017
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The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]

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Victor Dubowitz Honored at 2017 Researcher Meeting for Early Contributions to the SMA Field

August 23, 2017
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During the 2017 SMA Researcher Meeting, we honored one of our 2017 conference champions, Dr. Victor Dubowitz. Dr. Dubowitz’s early contributions to the SMA field […]

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Cure SMA-Funded Researchers, Diane Murrell and Deborah Boroughs, Publish Papers

August 17, 2017
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Two Cure SMA-funded projects have recently been published. Diane Murrell and her team published the paper, “The Experience of Families With Children With Spinal Muscular […]

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2017 Researcher Meeting Summary: Regulation of Splicing and SMN Protein Expression

August 15, 2017
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The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]

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2017 SMA Researcher Meeting Summary: SMA Therapy Development

August 10, 2017
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The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]

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2017 SMA Researcher Meeting Summary: Special Session on Fatigue and Muscle Weakness in SMA

August 7, 2017
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The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]

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Summer 2017 Issue of Compass Now Available

July 25, 2017
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The summer 2017 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced ten grants totaling $1.03 […]

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Summer 2017 Update to SMA Drug Pipeline is Released

July 1, 2017
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We’ve recently released an update to the SMA drug pipeline. This latest version includes: 19 active programs, including one approved therapy.  14 pharmaceutical partners.  5 […]

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Cure SMA Funded Standard of Care Paper Published in Neuromuscular Disorders Journal

June 30, 2017
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The journal Neuromuscular Disorders has published a paper reporting on the findings of an international workgroup convened to update the Standard of Care (SOC) for […]

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AveXis Announces Alignment with the FDA on Company’s GMP Commercial Manufacturing Process for AVXS-101

June 14, 2017
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AveXis today announced alignment with the U.S. Food and Drug Administration (FDA) on the company’s Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 following […]

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