News

September 17, 2015

Cure SMA Awards $150,000 to Dr. Lee Rubin at Harvard to Advance a New SMA Drug Discovery Program

Originally published on January 18, 2013.

Cure SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program, including drug discovery programs to make practical new therapies.   Dr. Rubin's project at Harvard University is the first of...

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September 17, 2015

Cure SMA and Nationwide Children’s Announce Multi-Million Dollar Award from NINDS to Advance CNS Gene Therapy for Spinal Muscular Atrophy

Originally published on March 13, 2013.

Nationwide Children’s Hospital (Columbus, OH) and Cure Spinal Muscular Atrophy (Elk Grove Village, IL) announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy...

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September 17, 2015

Pfizer Licenses Cure SMA Quinazoline Drug Program from Repligen

Originally published on January 3, 2013.

Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s spinal muscular atrophy (SMA) program, originally in-licensed from Cure SMA.

Cure SMA funded and directed the preclinical...

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September 17, 2015

Cure SMA Announces Grant to Cytokinetics for Preclinical Development of Tirasemtiv for Spinal Muscular Atrophy

Originally published on April 4, 2013.

Award to support testing of Tirasemtiv in mouse models of Spinal Muscular Atrophy.

Cytokinetics, Incorporated (Nasdaq: CYTK) and Cure SMA announced the award of a grant from Cure SMA to Cytokinetics to support...

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September 17, 2015

ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy

Originally published on April 23, 2013.

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced in April the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The study, which will begin enrolling patients soon, is a Phase 2 study in eight patients with infantile-onset SMA. The study,...

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September 17, 2015

Cure SMA Awards $150,000 to Dr. Burghes and Dr. Lorson to Investigate New Antisense Therapies for Spinal Muscular Atrophy

Originally published on May 2, 2013.

Cure SMA is dedicated to creating a treatment and cure for spinal muscular atrophy by funding and advancing a comprehensive research program, including drug discovery programs to make practical new therapies.  This is the third of four drug discovery project awarded by Cure...

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September 17, 2015

VALIANT Clinical Trial of Ambulatory Type III Spinal Muscular Atrophy Patients Conducted at Ohio State University Published in Muscle and Nerve

Originally published on May 21, 2013.

Results from the Phase II Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANT SMA) clinical trial led by Dr. John Kissel at The Ohio State University have been published in the journal...

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September 17, 2015

Cure SMA Awards $150,000 to Drs. Hua and Krainer at CSHL to Investigate Antisense Oligonucleotides for Spinal Muscular Atrophy

Originally published on August 8, 2013.

Cure SMA is dedicated to creating a treatment and cure for spinal muscular atrophy by funding and advancing a comprehensive research program, including drug discovery programs to make practical new therapies. This is the fourth drug discovery project funded by Cure SMA in 2013 with a...

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September 14, 2015

PTC Therapeutics Announces Selection of a Drug Development Candidate for Spinal Muscular Atrophy

Originally published on August 9, 2013.

PTC Therapeutics, Inc. (NASDAQ:PTCT) announced on August 8 the selection of a development candidate in its spinal muscular atrophy (SMA) collaboration with Roche and the SMA Foundation.

"We are excited about this important achievement in our SMA program", stated...

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September 14, 2015

Meet Newly Funded Cure SMA Researcher: Dr. Adrian Krainer

Originally published on August 15, 2013.

Dr. Krainer is a Professor at Cold Spring Harbor Laboratory, a member of the Cure SMA Scientific Advisory Board, and a long-term collaborator of Isis...

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