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 >
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 >
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 >
Status: Enrolling
Investigator: Ericka Greene
Study Coordinator:
Phone:
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 >
Status: Enrolling
Investigator: Ericka Greene
Study Coordinator: Sharon Halton
Phone: 713.441.3420
This is a Phase III, prospective, multicenter study with two study parts (epochs) to evaluate the efficacy, safety, and tolerability of HYQVIA/HyQvia (IGI, 10% with recombinant human hyaluronidase (rHuPH20) administered subcutaneously) for maint ... Read more >
