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About SMA: SMA Press

SMA Foundation and PTC Therapeutics Expand Partnership and Commit to an Initial Investment of Up to $60M to Advance Drug Discovery and Development Research in Regenerative Medicine

March 9, 2021

The Spinal Muscular Atrophy (SMA) Foundation and PTC Therapeutics have entered a new collaboration focused on regenerative medicine to further advance scientific research in SMA and other neuromuscular disorders. Recent FDA approvals of several disease modifying therapies for SMA has made regenerative medicine approaches the next frontier in drug discovery and development. The SMA Foundation-PTC…

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Scholar Rock Announces Positive Proof-of-Concept Data from TOPAZ Phase 2 Trial Interim Analysis of SRK-015 in Patients with Type 2 and Type 3 SMA

October 29, 2020

Scholar Rock announced positive six-month interim analysis results from the TOPAZ Phase 2 clinical trial yesterday. SRK-015 is a selective inhibitor of myostatin that has been shown to increase muscle mass and strength in animal models. Treatment with SRK-015 led to improvements in Hammersmith scale scores (primary efficacy endpoint) in all three cohorts of patients…

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Partnership between SMA Foundation, PTC Therapeutics, and Roche featured in article

September 28, 2020

Following the approval of Evrysdi, Roche released an article describing its partnership with the SMA Foundation and PTC Therapeutics to develop the drug. To read the article, please visit: https://www.roche.com/partnering/spinal-muscular-atrophy-sma-together.htm

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Arya Singh featured in New Haven Register article

September 20, 2020

Arya Singh, an SMA patient and Yale University student, was featured in a New Haven Register article on September 19th. In the article, Singh describes the challenges she faces as a patient and her experiences in clinical trials. To view the article, please visit: https://www.ctinsider.com/news/nhregister/article/This-Yale-student-won-t-let-a-rare-genetic-15579131.php?src=nhrhpcp To view a photo essay for the article, please visit:…

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SMA News Today publishes a series of articles on Evrysdi discovery and development

September 20, 2020

In August, SMA News Today published a series of articles on the discovery and development of Evrysdi, the first and only oral medicine for SMA approved by the FDA. The articles cover a range of topics including the role of the SMA Foundation in drug development, the experiences of patients taking Evrysdi, and the future…

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SMA featured in WuXi AppTec’s “Collaborations That Transform” webinar series

September 20, 2020

As part of its “Collaborations That Transform” webinar series, WuXi AppTec hosted three panels of distinguished SMA experts, representing patient, industry and physician perspectives. The panelists discussed the current state of SMA therapies and the promise of the next generation of treatment approaches. To view the webinar please visit: https://wxpress.wuxiapptec.com/webinar-sma/

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FDA approves Evrysdi (risdiplam) for treatment of Spinal Muscular Atrophy (SMA) in adults and children 2 months and older

August 7, 2020

The SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that Evrysdi™ (risdiplam) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA) in adults and children two months and older. Evrysdi showed clinically meaningful improvements in motor function across two clinical trials covering a broad range…

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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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Cerebrum podcast features SMA researcher Lee Rubin discussing recent advances in SMA therapeutics

April 5, 2019

SMA researcher and Harvard University professor Lee Rubin was featured in a podcast with Bill Glovin from the Dana Foundation to talk about the recent Cerebrum article he co-authored, “Spinal Muscular Atrophy: Huge Steps.” Both the article and podcast discuss recent advances in SMA therapeutics. To listen to the podcast, please visit: https://soundcloud.com/danafoundation/spinal-muscular-atrophy-huge-steps

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Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1

December 3, 2018

Basel, December 3, 2018 – Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of…

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Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of Spinal Muscular Atrophy

December 3, 2018

CAMBRIDGE, Mass., Nov. 27, 2018 (GLOBE NEWSWIRE) — Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the publication, “Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy” in the peer-reviewed journal Human Molecular Genetics. The publication details…

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