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

Research site

(Unclaimed)

Location

200 University Avenue East, Saint Paul, Minnesota, United States of America

Site insights

Top conditions

Top treatments

Trofinetide

Idebenone

NNZ-2566

Apitegromab

ION440

Nusinersen

trofinetide

Sarizotan

Cannabidiol

Steroids

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Data sourced from clinicaltrials.gov

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Active trials

4 of 47 total trials

A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)

In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will...

Active, not recruiting

Muscular Atrophy, Spinal

Drug: Nusinersen

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

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab (ONYX)

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

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

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