Showing 1 - 7 of 7 Muscle, Bone, and Cartilage Diseases & Disorders
A prospective, multi-center, observational study of the safety, tolerability and effectiveness of SPINRAZA in adult patients with Spinal Muscular Atr

Status: Open Not Enrolling

Investigator: Bing Liao

Study Coordinator: Anjana Singh

Phone: 713.441.9120

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

A Study to Evaluate CC-92480 in Combination With Carfilzomib and Dexamethasone (480Kd) Versus Carfilzomib and Dexamethasone (Kd) in Participants With Relapsed or Refractory Multiple Myeloma (SUCCESSOR-2)

Status: Enrolling

Investigator: Carrie Yuen

Study Coordinator: Danielle Sewall

Phone: 346.238.2674

The purpose of the study is to compare CC-92480 (BMS-986348) with carfilzomib and dexamethasone (480Kd) against carfilzomib and dexamethasone (Kd) in the treatment of RRMM. ... Read more >

Clinical Trial Readiness for SCA1 and SCA3

Status: Enrolling

Investigator: Tetsuo Ashizawa

Study Coordinator: Valerie Flores

Phone: 713.363.9803

The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments. Through US-European collaborations, the investigators will esta ... Read more >

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

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator: Anjana Singh

Phone: 713.441.9120

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

Long-Term, Observational, Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator: Aramide Balogun

Phone: 713.441.6955

Complement component 5 (C5) inhibition therapies have been available for patients with generalized Myasthenia Gravis (gMG) in the United States since the introduction of Soliris® in late 2017. Alexion is developing potential new therapies that ... Read more >

Myotonic Dystrophy Compilation of Clinical Findings

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator: Aramide Balogun

Phone: 713.441.6955

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: KaDeesia Brown

Phone: 713.441.9120

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 >