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7 recruiting studies matching your filters

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with…

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

Hoffmann-La RocheNCT05861999

Phase 1

An Open Label Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)

In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusiners…

Muscular Atrophy, Spinal

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pe…

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

Hoffmann-La RocheNCT05861986

Phase 3

An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy

In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about …

Muscular Atrophy, Spinal

A Phase 3, Randomized, Double-Blinded Study to Evaluate the Safety and Efficacy of Salanersen (BIIB115) After Onasemnogene Abeparvovec Treatment in Infants With Genetically Diagnosed Spinal Muscular Atrophy

In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about h…

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

BiogenNCT07444450

Spinraza (Nusinersen) SMA Pregnancy Exposure Study Within Existing SMA Registries

In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also…

Muscular Atrophy, Spinal

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dos…

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

Hoffmann-La RocheNCT05808764