News

August 3, 2018

2018 SMA Researcher Meeting Summary: SMA Therapy Development

2018 SMA Researcher Meeting Summary: SMA Therapy Development

The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA and seeks to create open communication of early, unpublished data to accelerate the pace of research. The meeting also furthers...

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Topics: Conference, Research, Front Page News

July 27, 2018

2018 SMA Researcher Meeting Summary: Clinical Research Studies for SMA

2018 SMA Researcher Meeting Summary: Clinical Research Studies for SMA

The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA and seeks to create open communication of early, unpublished data to accelerate the pace of research. The meeting also furthers...

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Topics: Conference, Research, Front Page News

July 25, 2018

2018 SMA Researcher Meeting Summary: Basic Research Podium Presentations

2018 SMA Researcher Meeting Summary: Basic Research Podium Presentations

The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA and seeks to create open communication of early, unpublished data to accelerate the pace of research. The meeting also furthers...

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Topics: Conference, Research, Front Page News

July 23, 2018

2018 SMA Researcher Meeting Special Session Summary: Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity

2018 SMA Researcher Meeting Special Session Summary: Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity

The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA and seeks to create open communication of early, unpublished data to accelerate the pace of research. The meeting also furthers...

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Topics: Conference, Research, Front Page News

July 12, 2018

Cure SMA Awards $300,000 Grant to Kevin Hodgetts, PhD, The Brigham and Women's Hospital

Cure SMA Awards $300,000 Grant to Kevin Hodgetts, PhD, The Brigham and Women's Hospital

Cure SMA has awarded a $300,000 preclinical drug discovery grant to Kevin Hodgetts, PhD, at the Brigham and Women’s Hospital, for his project, " Pre-Clinical Development of LDN-5178 for the Treatment of SMA.”

This grant will be conducted by two academic research teams...

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Topics: Our Impact, Research, Front Page News

July 11, 2018

Dr. John Kissel, Chair of Neurology at OSU Wexner Medical Center, Retires After 40 Years

Dr. John Kissel, Chair of Neurology at OSU Wexner Medical Center, Retires After 40 Years

Dr. John Kissel has treated patients with neuromuscular disorders from all over the United States. He specializes in spinal muscular atrophy and has been instrumental in his practice at one of the country's leading centers in neuromuscular medicine.

Dr. Kissel’s interest in SMA...

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Topics: Support & Care, Research, Front Page News

July 10, 2018

Roche Releases Community Update on Risdiplam

Roche Releases Community Update on Risdiplam

The following is a community update from Genentech/Roche on the risdiplam (RG7916) program.

Dear SMA advocacy community,

As requested, we are very pleased to share an update on the RG7916 program.

We would also like to extend a special thanks to study participants and...

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Topics: Clinical Trials, Research, Front Page News

July 10, 2018

Cure SMA Awards $300,000 Grant to Umrao Monani, PhD, Columbia University

Cure SMA Awards $300,000 Grant to Umrao Monani, PhD, Columbia University

Cure SMA has awarded a $300,000 preclinical drug discovery grant to Umrao Monani, PhD, at Columbia University, for his project, " Restoring function at the NMJ: A novel means to treat SMA.”

A drug directed at SMN enhancement, Spinraza, has recently become available but is...

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Topics: Our Impact, Research, Front Page News

June 25, 2018

Summer 2018 Issue of Compass Now Available

Summer 2018 Issue of Compass Now Available

The summer 2018 issue of Compass is now available online.

As part of our most recent round of funding, we’ve announced ten grants totaling $1,325,000 million for basic research.

This issue of Compass...

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Topics: Our Impact, Research, Front Page News

June 19, 2018

PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings

On Saturday, PTC Therapeutics, Inc. announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy...

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Topics: Clinical Trials, Research, Front Page News

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