News

August 18, 2016

Cure SMA Plans a Series of Educational Modules for the SMA Community

Cure SMA Plans a Series of Educational Modules for the SMA Community

With the recent news that Biogen and Ionis will submit nusinersen for FDA approval—along with other recent developments, including the breakthrough designation for AveXis’s...

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Topics: Community & Awareness, Our Impact, Clinical Trials, Advocacy, Front Page News

August 17, 2016

Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy

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Topics: Research, Advocacy, Front Page News

July 14, 2016

Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports

Dr. Rashmi Kothary and his team have recently published a paper, “Differential induction of muscle atrophy pathways in two mouse models of spinal muscular atrophy” in the journal Scientific Reports. This research was funded in part by grant from Cure...

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Topics: Advocacy, Research, Front Page News

June 24, 2016

Cure SMA Launches New Surveys to Share Our Community’s Experience

Cure SMA Launches New Surveys to Share Our Community’s Experience

Cure SMA is committed to addressing the needs of individuals and families affected by SMA. In order to accomplish this goal, we’ve been working for the past several years to collect data and information on the experiences of living with SMA, as well as our community’s goals, hopes...

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Topics: Support & Care, Our Impact, Advocacy, Front Page News

June 22, 2016

Cure SMA Posts Keynote Presentations on the NDA Process

Cure SMA Posts Keynote Presentations on the NDA Process

The ultimate goal in drug development is to get treatments approved for as many people as possible, as quickly as possible. Because these issues are complex, we want our community to be knowledgeable about the burden of proof required to support an FDA-approved treatment, and...

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Topics: Our Impact, Conference, Advocacy, Front Page News

June 1, 2016

2016 Update on SMA-FDA Interactions

2016 Update on SMA-FDA Interactions

The SMA drug pipeline has grown dramatically in just over a decade. Of the 18 programs in the pipeline, six of these are in clinical trials, and several of those are in Phase 2 and Phase 3 clinical trials.

Reaching and achieving FDA approval is a major step in our path to a treatment...

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Topics: Our Impact, Advocacy, Front Page News

April 20, 2016

Cure SMA and the SMA Community Featured by Astellas Pharmaceuticals

Cure SMA and the SMA Community Featured by Astellas Pharmaceuticals

Cure SMA is featured by Astellas Pharmaceuticals as part of their new Changing Tomorrow Together website. Cure SMA Research Director Jill Jarecki contributed a

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Topics: Community & Awareness, Advocacy, Front Page News

April 8, 2016

Global Genes Features Cure SMA in Patient Voice Project

Global Genes Features Cure SMA in Patient Voice Project

Global Genes recently published, “From Molecules to Medicines: How Patients Can Share Their Voices Through the Drug Development Process.” This

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Topics: Community & Awareness, Advocacy, Front Page News

March 10, 2016

Senate HELP Committee Passes Another 7 Innovation Bills

Senate HELP Committee Passes Another 7 Innovation Bills

The Senate Health, Education, Labor and Pensions (HELP) Committee yesterday passed an additional 7 bills out of committee. Along with the 7 bills previously passed out of committee, that makes 14 bills in total...

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Topics: Advocacy, Front Page News

February 9, 2016

Senate HELP Committee Passes Innovation Bills

Senate HELP Committee Passes Innovation Bills

The Senate Health, Education, Labor and Pensions (HELP) Committee has passed 7 bills out of committee as part of its “step-by-step” approach to Innovation and 21st Century Cures. Among them is The...

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Topics: Advocacy, Front Page News

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