Cytokinetics and Astellas Release Further Details on Upcoming Phase 2 Trial

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Cytokinetics, in partnership with Astellas, has officially announced that recruitment will begin in the second half of 2015 for a Phase 2 clinical trial testing CK-2127107, a muscle activator. This randomized, double-blind study is expected to enroll 75 patients with SMA type II, III, or IV. Patients will be dosed for four weeks, and will […]

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Cure SMA Joins The Research Acceleration and Innovation Network

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Cure SMA has become a participating organization of The Research Acceleration and Innovation Network (TRAIN), a program of FasterCures, bringing the total number of venture philanthropies in TRAIN to 80. FasterCures established TRAIN in 2005 to connect disruptive innovators in the disease research space with the vital resources, tools and relationships to catalyze development of

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2015 Family Friendly Poster Session Presenters Announced

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The Family Friendly Research Poster Session is one of the most anticipated events of our Annual SMA Conference. Each year, Cure SMA invites researchers from the SMA Researcher Meeting, which is held in parallel with the family conference, to present their findings to families in way that is accessible to them. During the event, families

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Update on Clinical Development of RG7800

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F. Hoffman-La Roche, PTC and the SMA Foundation recently provided an update on the clinical development of RG7800, an investigational SMN2 splicing modifier that is being studied as a potential treatment for spinal muscular atrophy (SMA): “In November 2014, Roche initiated the Moonfish trial (Phase 1b/2a) based on supportive results from a trial in healthy

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Cure SMA Funds $1.85 Million in New Research Grants in 2014-2015

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Over the past few months, Cure SMA has announced a total of $1.85 million in new research funding. Our model is designed to fund both a breadth and a depth of projects, in order to continuing building up both a breadth and depth of programs in the drug pipeline. This includes funding for: Basic research,

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Cure SMA Announces Additional $445,000 for Nationwide Children’s Hospital Gene Therapy Program led by Brian Kaspar, PhD

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Cure SMA, the leading organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently provided an additional $445,000 grant toward a gene therapy for SMA at Nationwide Children’s Hospital. Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at Nationwide Children’s, and his team have made promising strides toward therapies

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New 21st Century Cures Discussion Draft Released

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The US House of Representatives Energy and Commerce Committee has released a new 21st Century Cures discussion draft. This is a revised and expanded version of the first draft that was released in January. The 21st Century Cures initiative was created to investigate how the government can help “accelerate the discovery, development, and delivery of

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Registration for the 2015 Annual SMA Conference Closes in Two Weeks

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The deadline to register for our Annual SMA Conference is just two weeks away. Registration will close on Friday, May 9. Over 1,000 people have already registered—don’t miss your chance to be part of the action! The Annual SMA Conference is the largest SMA conference in the world for families, researchers, and medical providers. The

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Cure SMA Publishes New Care Series Booklet on Musculoskeletal System

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Cure SMA has recently released an updated version of our latest care series booklet, The Musculoskeletal System. As with all our care series booklets, The Musculoskeletal System was written by medical professionals who specialize in SMA, and reviewed by families to ensure that it is clear and easy to understand, and that it addresses the

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