Trial Search Results
Clinical Study of Spinal Muscular Atrophy
The investigators propose to prepare for clinical trials where SMA patients are asked to join the research effort. The visits will include questions, physical exam, blood drawing, and sometimes X-rays and a skin biopsy. The investigators will use modern computer methods to process the information during which the investigators will plan a clinical trial. Once the clinical trial begins, the investigators will offer SMA patients participation if they meet the criteria for that trial.
Identifying an effective SMA treatment is very important because there is currently none. Clinical trials are the only way to decide whether a new treatment works in SMA patients or not.
Stanford is currently not accepting patients for this trial.
Collaborator: The Spinal Muscular Atrophy Foundation
- Clinical diagnosis of Spinal Muscular Atrophy
- Genetic diagnosis of SMN gene deletion
- Parents or if applicable subjects must give informed consent
- must be capable of complying with the study procedures
- Female subjects of child-bearing potential must agree to undergo pregnancy test prior
to radiological studies
- Diagnosis of SMA before age 19 years
- Unstable medical condition precluding participation
- Significant respiratory compromise that would interfere with safe travel to site of
evaluation. (The clinical site PI decides when air travel is not recommended and when
the patient's location is not within a reasonably safe driving distance (upper limit
Ages Eligible for Study
N/A - N/A
Genders Eligible for Study