The International Spinal Muscular Atrophy Patient Registry (the Registry) was founded in 1986 at Indiana University. This project is supported by the Patient Advisory Group of the International Coordinating Committee for SMA Clinical Trials (ICC) which includes Families of SMA, FightSMA, the Muscular Dystrophy Association, and the Spinal Muscular Atrophy Foundation, and other SMA advocacy groups. The ICC is a volunteer committee composed of stakeholders from the SMA community who work together to address the opportunities and challenges associated with effectively organizing clinical trials of new treatments for SMA.

The Registry functions as a liaison between patients and families interested in participating in research and researchers interested in studying SMA. The Registry contains information from over 1,600 families and over 1,700 individuals with SMA from all over the world and continues to grow. The Registry has helped recruit participants for clinical trials and has provided data for 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.

Participant information is stored in a secure database. Researchers who are interested in studying SMA can request two types of data from the Registry, de-identified information and identifiable information. De-identified information does not contain any names or personal identifiers, and can be given to researchers without having to contact Registry families. Identifiable information includes information that can identify you and will never be released without getting your written permission to do so. Identifiable information includes data such as names and dates of birth. Some researchers may also request contact with families to obtain specific information or to request participation in a research study. In these instances, the Registry will contact each potential participant to ask if they are willing to share their identifiable information for a research project.

The Registry recognizes the importance of global collaboration. Working together with researchers from all over the world is very important in the investigation of SMA. In 2008 the Registry joined the group, Translational Research in Europe for the Assessment and Treatment of Neuromuscular Diseases (TREAT-NMD) in a global collaboration to further the research goals of the neuromuscular disease community. TREAT-NMD has developed a global database that compiles deidentified data transferred from participating registries around the world. The main objective of the TREAT-NMD database is to assess the feasibility of clinical trials, to facilitate the planning of clinical trials and to support the enrollment of patients in clinical trials. This international database will also be used to answer questions regarding the prevalence of particular neuromuscular disorders and assessing the support of other activities such as assessing standards of care and diagnosis.

Researchers who wish to contact members of the TREAT-NMD data set will submit a proposal to TREAT-NMD.  Subjects who meet the inclusion criteria for the clinical trial based on information contained in the TREAT-NMD database. If a subject meets the inclusion criteria the Registry from which the data was transferred will be notified. The Registry will then be responsible for notifying participants of their eligibility for a particular clinical trial. TREAT-NMD will not have access to any identifiable subject information or direct access to any subjects. Subjects who are eligible for a particular study can only be contacted through the appropriate national Registry.