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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

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

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...

Active, not recruiting

Rett Syndrome

Genetic: TSHA-102