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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 >
Status: Open Not 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 >
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 >
Status: Open Not Enrolling
Investigator: Ericka Greene
Study Coordinator: Aramide Balogun
Phone: 713.441.6955
The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks. Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at ... Read more >
Status: Enrolling
Investigator: Ericka Greene
Study Coordinator: KaDeesia Brown
Phone: 713.441.9120
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: Open Not Enrolling
Investigator: Ericka Greene
Study Coordinator: Aramide Balogun
Phone: 713.441.6955
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 >