International SMA Patient Registry
International SMA Patient Registry
This is a registry of individuals affected by Spinal Muscular Atrophy (SMA) and/or family
 members of individuals affected by SMA. The purpose of the registry is to allow researchers
 studying the biological basis of SMA and potential therapies in SMA access to individuals
 interested in participating in research and/or experimental therapies. The International SMA
 Patient Registry is supported by CureSMA.
This project is supported by CureSMA (http://curesma.org/).

 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 2,500 families and over 3,000 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.

 Individuals and families affected by SMA are invited to join the Registry. Participants are
 asked to complete questionnaires about the symptoms, treatment, medications, and other
 experiences with SMA. Visit the Participant Portal for more information on how to sign up for
 the Registry.

 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.
Inclusion Criteria:

 -  Individuals or family members of individuals who have been diagnosed with SMA.