Learn more orphan products.
Research helps us better understand diseases and can lead to advances in diagnosis and treatment. This section provides resources to help you learn about medical research and ways to get involved.
Support and advocacy groups can help you connect with other patients and families, and they can provide valuable services. Many develop patient-centered information and are the driving force behind research for better treatments and possible cures. They can direct you to research, resources, and services. Many organizations also have experts who serve as medical advisors or provide lists of doctors/clinics. Visit the group’s website or contact them to learn about the services they offer. Inclusion on this list is not an endorsement by GARD.
These resources provide more information about this condition or associated symptoms. The in-depth resources contain medical and scientific language that may be hard to understand. You may want to review these resources with a medical professional.
NIH Hosts Rare Disease Day Event, Twitter Chat
January 24, 2019
Clinical Trials Bring Hope to Kids with Spinal Muscular Atrophy
November 29, 2017
FDA Approves First Drug for Spinal Muscular Atrophy
January 11, 2017
Questions sent to GARD may be posted here if the information could be helpful to others. We remove all identifying information when posting a question to protect your privacy. If you do not want your question posted, please let us know. Submit a new question
If members of a couple are carriers of SMA and had a baby affected with SMA type 1, is there a chance that they could have babies affected with other types of SMA or it will always be the same type that would appear in their children? See answer
Are there any other diseases or conditions that have similar symptoms to SMA, or mimic the symptoms of SMA? See answer