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SMA Press


About SMA: SMA Press

Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with Type 1 SMA

January 23, 2020

The SMA Foundation’s partners, Roche and PTC Therapeutics, announced positive data from the pivotal Part 2 of FIREFISH, a study evaluating risdiplam in infants aged 1-7 months with Type 1 SMA. The study met its primary endpoint of a statistically significant proportion of infants sitting without support for at least five seconds after one year…

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Scholar Rock announces completion of TOPAZ trial enrollment

January 10, 2020

Scholar Rock announced today that enrollment of its TOPAZ Phase 2 trial of SRK-015 in patients with Type 2 and Type 3 SMA is complete. SRK-015 is a selective inhibitor of myostatin that has been shown to increase muscle mass and strength in animal models. For more information, please see the press release from Scholar…

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Roche Initiates New Phase 2 Clinical Trials

September 27, 2016

Roche has announced that the company is initiating two new Phase 2 studies in patients with Spinal Muscular Atrophy (SMA) to evaluate RG7916—SUNFISH and FIREFISH. RG7916 is an orally available drug that directly targets the underlying molecular deficiency of SMA by modulating SMN2 splicing to increase expression of full-length SMN2 mRNA and SMN protein from…

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Bloomberg Article: New Hope for Kids With a Rare Disease, Thanks to a Dinner Party

September 2, 2016

Since grade school, Lauren Gibbs had been a dogged defensive player on her wheelchair-basketball team. Offense wasn’t a big part of her game because it was a struggle to heave the ball high enough to hit the rim. Then Gibbs, who was born with spinal muscular atrophy, enrolled in a trial of an experimental drug…

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Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy

August 1, 2016

CAMBRIDGE, Mass. & CARLSBAD, Calif.–Aug. 1, 2016– Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) today announced that nusinersen, their investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint pre-specified for the interim analysis of ENDEAR, the Phase 3 trial evaluating nusinersen in infantile-onset (consistent with Type 1) SMA. The analysis found that infants receiving…

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Update on LMI070 for the Treatment of Type 1 SMA

July 25, 2016

In May, we shared with you news of the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). This decision was made because results from an animal study, using daily dosing for a year compared to weekly dosing in the human study, showed unexpected…

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AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for SMA Type 1

July 20, 2016

Chicago,Ill., July 20, 2016 – AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1…

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Letter to the SMA Community on the Study of LMI070 for the Treatment of Type 1 SMA

June 1, 2016

Wednesday May 25th, 2016 – Letter from Novartis: We have made the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). Animal safety studies were taking place in parallel with this trial, which is a common practice when studying potentially life threatening diseases to…

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FightSMA Announces First-Time Inclusion of Spinal Muscular Atrophy in Defense Department’s Peer-Reviewed Medical Research Program

June 1, 2016

WASHINGTON, D.C., May 26, 2016–FightSMA is proud to announce that, for the first time, the U.S. Senate Appropriations committee has included spinal muscular atrophy as a research topic in the Defense Department’s Peer-Reviewed Medical Research Program as part of the Fiscal Year 2017 Defense Appropriations bill. This exciting news comes as the result of months…

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Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development

January 7, 2016

-Phase 1 Study Initiated in Healthy Volunteers with New SMN2 Splicing Modifier – RG7916 – SOUTH PLAINFIELD, N.J., Jan. 7, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (Nasdaq: PTCT) today announced that RG7916, an additional SMN2 splicing modifier from the company’s joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA), has…

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Data from Ongoing Study of AVXS-­101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress

October 5, 2015

Brighton, United Kingdom. October 5, 2015 – AveXis, Inc., a clinical stage gene therapy company developing treatments for rare and life-­threatening neurological genetic diseases, presented data from its ongoing Phase 1/2 trial of AVXS-­101, the company’s initial product candidate for the treatment of spinal muscular atrophy (SMA) Type 1, at the International Congress of the…

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June 11, 2015

Webcast to review study data scheduled for Thursday, June 11 at 8:30 a.m. Eastern Time CARLSBAD, Calif., June 11, 2015 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA). Previously the company reported data from…

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