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

June 16, 2018

Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference

Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference

Today, Cytokinetics announced data from the Phase 2 clinical study of reldesemtiv in patients with SMA, presented in an oral presentation by...

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Topics: Clinical Trials, Research, Front Page News

June 14, 2018

Biogen to Present New Data at the 2018 Annual SMA Conference

Biogen to Present New Data at the 2018 Annual SMA Conference

Yesterday, Biogen announced it will present data from its SPINRAZA clinical development program –the largest of its kind, with more than six years of...

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Topics: Conference, Research, Front Page News

June 1, 2018

Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015

Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015

Scholar Rock, a biotechnology company based in Boston, recently announced that it has completed dosing of the first cohort and is continuing...

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Topics: Clinical Trials, Research, Front Page News

May 30, 2018

Roche Releases Community Statement on Olesoxime Program

Roche Releases Community Statement on Olesoxime Program

Roche today provided the following community statement on olesoxime.

Dear members of the SMA Community,

We would like to provide you with an update about olesoxime, an investigational molecule in SMA.

Since we bought olesoxime from Trophos in 2015, we have had many...

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Topics: Clinical Trials, Research, Front Page News

May 24, 2018

Cure SMA Awards $150,000 Grant to Kathryn Swoboda, MD, Massachusetts General Hospital

Cure SMA Awards $150,000 Grant to Kathryn Swoboda, MD, Massachusetts General Hospital

Cure SMA has awarded a $150,000 research grant to Kathryn Swoboda, MD, at the Massachusetts General Hospital, for her project, "Clinical and pathologic correlations in patients with early infantile-onset SMA.”

SMA is caused by mutations in the SMN1 gene resulting in low levels of...

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Topics: Our Impact, Research, Front Page News

May 22, 2018

Biogen Releases Community Statement on Spinraza Access and New Data

Biogen Releases Community Statement on Spinraza Access and New Data

Biogen has released the following community statement on Spinraza access and new research data.

Dear Members of the SMA community,

Thank you for your continued support, efforts to raise awareness about SMA, and participation in clinical trials and studies as we seek to expand...

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Topics: Clinical Trials, Research, Front Page News

May 15, 2018

Novartis Successfully Completes Acquisition of AveXis, Inc.

Novartis Successfully Completes Acquisition of AveXis, Inc.

Dear SMA Community, 

Following the announcement last month, we wanted to let you know that AveXis (the gene therapy company developing a new approach to treat SMA known as AVXS‐101) is

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Topics: Research, Front Page News

May 8, 2018

Cure SMA Awards $150,000 Grant to Chad Heatwole, MD, University of Rochester

Cure SMA Awards $150,000 Grant to Chad Heatwole, MD, University of Rochester

Cure SMA has awarded a $150,000 research grant to Chad Heatwole, MD, at the University of Rochester, for his project, " Development of a Clinically Relevant Outcome Measure for Pediatric SMA Therapeutic Trials.”

Dr. Heatwole and his team are working to develop SMA-specific...

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Topics: Our Impact, Research, Front Page News

May 1, 2018

Invitae Partners with Biogen to Offer Free Genetic Testing for SMA

Invitae Partners with Biogen to Offer Free Genetic Testing for SMA

Invitae Corporation recently announced that it will partner with Biogen to offer genetic testing at no charge to patients who may have...

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Topics: Support & Care, Research, Front Page News

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