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Gillette Children's Specialty Healthcare | St. Paul Campus - Orthopaedic Research

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Active trials

4 of 45 total trials

A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec (RESPOND)

The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atr...

Active, not recruiting
Muscular Atrophy, Spinal
Drug: Nusinersen

This Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spina...

Active, not recruiting
Anti-myostatin
Neuromuscular Manifestations
Drug: Placebo
Drug: Apitegromab

The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Ty...

Active, not recruiting
Anti-myostatin
Neuromuscular Manifestations
Drug: Apitegromab

The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene the...

Enrolling
Rett Syndrome
Genetic: TSHA-102

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Acadia Pharmaceuticals logo
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