Roche Provides New Update on Clinical Development of RG7916

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Roche, PTC Therapeutics, and the SMA Foundation have released a community update on the development of their SMN2 splicing modifier clinical development program: Dear SMA community, Roche, together with our collaboration partners PTC Therapeutics and the SMA Foundation, would like to provide you with an update on our SMN2 splicing modifier clinical development program. Please […]

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2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function

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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 The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research.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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Cure SMA Welcomes Aboard New Board and Committee Members

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Recently, Cure SMA confirmed three new board members and one new committee member. These new members will provide additional leadership in the areas of advocacy, FDA/regulation, and research. We extend a warm welcome to new board members Kelly Cole; Doug Kerr, MD, PhD, MBA; and Thomas Murray, PhD. We also extend a warm welcome to

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2016 SMA Researcher Meeting Summary: SMA Therapy Development

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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 The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research.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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We’re Partnering With The Mighty!

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We’re thrilled to announce a new partnership that will bring Cure SMA’s resources in front of The Mighty‘s wide-reaching readership. We will now have a growing home page on The Mighty and appear on many stories on the site, allowing us to get many more people involved with our organization. The Mighty is a story-based

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2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 2

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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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2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 1

Leave a Comment / Clinical Trials, Conference, Front Page News, Research /

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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Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene

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During the 2016 SMA Researcher Meeting, the anniversary of the identification of the SMN genes by Judith Melki MD, PhD (Inserm, Universite Paris) was recognized. Genetic studies of families with a history of SMA allowed researchers to localize the region containing the gene responsible for SMA to the long arm of chromosome 5 in 1992.

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2016 SMA Researcher Meeting Summary: Clinical Research Studies

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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. This update covers a session on clinical research and outcome measures. The goal of this session is to present the results of important studies that could influence clinical trial design

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