The SMA Foundation has worked in partnership with several other Spinal Muscular Atrophy organizations to establish national and international networks of clinics ready for clinical trials. Through regional networks in the U.S. like the Pediatric Neuromuscular Clinical Research (PNCR) Network and Project Cure, and international organizations such as the International Coordinating Committee (ICC) and TREAT-NMD, the SMA community has collaborated effectively on recruiting patients, establishing standards of care, standardizing outcome measures, and supporting multi-site clinical efforts, such as the Biomarkers for SMA (BforSMA) and the PNCR Network Natural History studies.
International Coordinating Committee for Clinical Trials
The International Coordinating Committee for Clinical Trials in SMA (ICC) is a volunteer committee composed of families and patients, clinicians, advocates, industry, and regulatory agencies from the SMA community. The ICC’s mission is to facilitate the conduct of efficient and successful clinical trials in SMA that lead to new treatments and ultimately improve care for people living with SMA. ICC members work to fulfill their mission, in part, by improving or developing new outcome measures for SMA. In addition, the Standard of Care Subcommittee of the ICC researched and developed a set of guidelines for patient care. This project resulted in the publication of the Consensus Statement on Standard of Care for SMA in Child Neurology and was followed up by the development and publication of the Family Guide to the Consensus Statement, available in both English and Spanish.
The International Spinal Muscular Atrophy Patient Registry was founded in 1986 at Indiana University. The Registry connects patients and families interested in participating in research, and researchers interested in studying SMA. The Registry contains information from more than 1,600 families and over 2,400 individuals with SMA from all over the world and continues to grow. The Registry has helped recruit participants and provide data for clinical trials and other important SMA research studies. The Registry helps centralize information on this rare genetic disease, provides families a way to learn about research studies, and provides researchers a way to find research participants.