Clinical Study of Spinal Muscular Atrophy (SMA)
The Pediatric Neuromuscular Clinical Research Network for SMA is conducting a clinical study of SMA. The purpose of this study is to learn more about the natural history of SMA and to obtain information needed to plan and prepare for a clinical trial for SMA. We hope to initiate a clinical trial for SMA in the near future.
We're looking for patients of any age with SMA diagnosed before the age of 19 years. While we are looking to recruit patients with all clinical types of SMA, we can only enroll patients with a deletion of exon 7 and those stable enough to travel to one of the PNCR clinical sites for study visits.
PNCR Clinical Sites
This study of SMA is being conducted at three clinical sites on the east coast:
- Columbia University Medical Center (New York City)
- Children's Hospital (Boston)
- The Children’s Hospital of Philadelphia (Philadelphia)
Participants will be evaluated at one of these clinics regularly at no charge. During each visit, participants undergo several tests. Participants are expected to travel to their enrolling clinic every 2 months for the first 6 months, every 3 months for the next 6 months, and every 6 months thereafter, for a period of 36 months.
If you have any questions or comments about this study, or to find out whether you or your child qualifies, please contact Jackie Montes:
- Jackie Montes at (212) 342-5767 or email