Showing 1 - 3 of 3 Muscular Dystrophy
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:

Phone:

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

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

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator:

Phone:

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:

Phone:

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 >