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

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Trofinetide
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trofinetide
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Active trials

3 of 47 total trials

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

The primary purpose of this study is to evaluate the safety and tolerability of ION440.

Enrolling
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Procedure: Sham procedure
Drug: ION440

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...

Active, not recruiting
Rett Syndrome
Genetic: TSHA-102

Trial sponsors

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Acadia Pharmaceuticals logo
Biogen logo
Allergan logo
Ionis Pharmaceuticals logo
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Santhera logo
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University of Washington logo
Ann & Robert H Lurie Children's Hospital of Chicago logo

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