Showing 1 - 9 of 9 Muscle, Bone, and Cartilage Diseases & Disorders
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

Status: Enrolling

Investigator: Sheetal Shroff

Study Coordinator:

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The purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG). NCT number: NCT03971422 ... Read more >

A Prospective, Multicenter Study to Evaluate the ConforMIS iTotal® (CR) Knee Replacement System

Status: Open Not Enrolling

Investigator: Terry Clyburn

Study Coordinator: Rosbel Brito

Phone: 918.813.7873

This study is designed to observe the long term clinical outcomes of patient-specific knee arthroplasty in patients with osteoarthritis. ... Read more >

A prospective, multi-center, observational study of the safety, tolerability and effectiveness of SPINRAZA in adult patients with Spinal Muscular Atr

Status: Enrolling

Investigator: Jason Thonhoff

Study Coordinator:

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The purpose of this study is to measure the safety and effectiveness of Spinraza in adults with SMA. ... Read more >

A Randomized, Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

Status: Enrolling

Investigator: Sheetal Shroff

Study Coordinator:

Phone:

The purpose of the MycarinGstudy is to evaluate the long-term safety, tolerability and long-term efficacy of rozanolixizumab in study participants with generalized myasthenia gravis (MG). NCT number: NCT04124965 ... Read more >

An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients With Congenital Myasthenic Syndrome (CMS)

Status: Enrolling

Investigator: Sheetal Shroff

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The primary objective of the study is: • To provide patients with CMS access to amifampridine phosphate therapy until the product becomes commercially available or development is discontinued. The secondary objective of the study is: • ... Read more >

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1: END-DM1

Status: Enrolling

Investigator: Ericka Greene

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Provide natural history data, biomarkers, and biological understanding of the phenotypic heterogeneity of myotonic dystrophy type 1 (DM1). ... Read more >

Myotonic Dystrophy Compilation of Clinical Findings

Status: Open Not Enrolling

Investigator: Ericka Greene

Study Coordinator: Sharon Halton

Phone: 713.441.3420

The purpose of this protocol is to make available clinical findings, including blood pressure, pulmonary function tests, cardiac function tests, clinical evaluations, cognitive evaluations, performance tests, medications, routine blood work eval ... Read more >

Neuromuscular Observational Research (MOVR) Data Hub Protocol

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator:

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This is a registry of people with neuromuscular diseases. It is the intent to follow patients with ALS and other neuromuscular diseases to determine best practices for care delivery. ... Read more >

Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator:

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The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG). [NCT03920293] ... Read more >