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Spinal Muscular Atrophy (SMA)
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Spinal Muscular Atrophy (SMA) trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Spinal Muscular Atrophy (SMA) trials may be worth asking aboutNorth America
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Background: SBMA is an inherited chronic disease. It affects males in mid to late adulthood. It causes slowly progressive weakness of muscles and hand tremors.…
Background: Androgen effects in humans are usually (but not always) mediated by the androgen receptor which is coded for by the androgen receptor gene (AR gene…
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with…
In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about ho…
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about h…
This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it mo…
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study is an extension…
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with S…