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A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma (ARISER)

• Source: A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER

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Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

• Authors : Genentech, Inc.

• Source: Pilot Study Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

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Newborn Screening for Spinal Muscular Atrophy (SMA-NBS)

• Authors : Novartis Gene Therapies; Hoffmann-La Roche; Oxford University Hospitals NHS Trust

• Source: Population-based New-Born Screening of Spinal Muscular Atrophy to Evaluate the Uptake and Feasibility in the UK Context

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Early Check: Expanded Screening in Newborns

• Authors : University of North Carolina, Chapel Hill; The John Merck Fund; Duke University

• Source: Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in NewbornsFinkel, Mercuri, Darras, Kuntz, Kirschner et al, 2017Birnkrant, et al, 2018Winarni,

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Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study (SMA-AtHome)

• Authors : Roche Pharma AG

• Source: Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study

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