September 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 total investment of […]

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2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 1

We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers a session on SMN Partners and Therapeutic Targets. The session was moderated by Arthur Burghes, MD, PhD. SMN Partners and Candidate Therapeutic Targets, Part 1 Individuals with

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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 Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “This

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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 Pharmaceuticals.  The grant award to Dr. Krainer’s lab is the fourth drug discovery project funded by Cure SMA in 2013 with a total investment

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Cure SMA Scientific Advisory Board Highlights Findings from The 2013 SMA Research Group Meeting

Originally published on August 28, 2013. The Cure SMA Scientific Advisory Board (SAB) helps organize, select the content, and chair sessions at the Cure SMA Research Group Meeting. Our SAB has written a summary of the scientific findings from the 2013 meeting.   During the 2013 meeting, 110 research presentations were given, including 38 talks during

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Meet Newly Funded Cure SMA Researchers: Oscar Mayer, MD PhD and Vanessa Battista, MS

Originally published on September 10, 2013. Dr. Mayer and Ms. Battista were awarded funding by Cure SMA for one of four new clinical care focused projects. The project is being conducted at the Children’s Hospital of Philadelphia. This award for $50,000 will fund a pilot study to assess the decision making process by parents of

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Meet Newly Funded Cure SMA Researcher: Dr. Kathryn Swoboda

Originally published on September 17, 2013. Dr. Swoboda was awarded funding by Cure SMA for one of four Clinical Care focused projects. The project is being conducted at the University of Utah. This award for $50,000 will fund a study to determine whether children/preadolescents with SMA type II demonstrate impaired glucose tolerance after glucose loading

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Meet Newly Funded Cure SMA Researcher Dr. Matthew A. Halanski

Originally published on September 25, 2013. Dr. Matthew Halanski was awarded funding by Cure SMA for one of four Clinical Care focused projects. The project is being conducted at the University of Wisconsin. This award for $50,000 will fund a project to create a database of patients with SMA from multiple clinical centers.  It will

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