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Onasemnogene Abeparvovec Effective in Treatment of Older, Heavier Children with SMA

December 26, 2023

A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1.

United Kingdom United States Simon Dannhauer Lancet Regional Health Philadelphia Infant Test Neuromuscular Disorders

Source: ajmc.com

Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial

September 19, 2023

Topline data from Phase 3 trial expected in Q4 2024 Company to present encore 36-month efficacy, safety, and patient-reported outcomes data from the Phase 2 TOPAZ trial extension at the 2023...

North Charleston South Carolina United States Jay Backstrom Thomaso Crawford Johns Hopkins University

Source: marketscreener.com

SMA in Adults: Interim Data Back Nusinersen Efficacy

April 27, 2023

New trial focuses on drug's performance in spinal muscular atrophy beyond childhood

University Of Rochester New York United States John Gever Health Index National Institute Of Neurological Disorders

Source: medpagetoday.com

New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA - Scholar Rock Holding (NASDAQ:SRRK) - Press Release

October 22, 2022

- Tertiary endpoint data show trends of continuous improvement in activities of daily living, fatigue, and endurance over 24 months - These data indic

United States Comunidad Autonoma De Cataluna Jay Backstrom Basil Darras Scholar Rock Inc International Scientific Congress

Source: wiredprnews.com

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