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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.
Clinical Trials Bring Hope to Kids with Spinal Muscular Atrophy
November 29, 2017
FDA Approves First Drug for Spinal Muscular Atrophy
January 11, 2017
18th Annual International Spinal Muscular Atrophy Research Group Meeting
Thursday, June 12, 2014 -
Saturday, June 14, 2014
Location: Gaylord National , National Harbor, MD
The overall goal of the meeting is to provide a venue for researchers to share unpublished data and to develop collaborations, with the intent of accelerating the discovery of a treatment for this devastating disease. In addition, the specific aims for the conference are: 1.To enable open communication of early, unpublished scientific data, accelerating the pace of research. 2.To provide a forum to discuss timely topics in SMA openly with the entire research community. 3. To create a sense of community among SMA researchers, resulting in productive research partnerships.
Contact: Dr. John D. Porter(301) 496-5739 firstname.lastname@example.org
Co-funding Institute(s): National Institute of Neurological Disorders and Stroke, Office of Rare Diseases Research
The following diseases are related to Spinal muscular atrophy. If you have a question about any of these diseases, you can contact GARD.
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
My brother's daughter was recently diagnosed with spinal muscular atrophy. What can you you tell me about this condition, particularly as it relates to the SMN genes? Can this condition be treated? What is the prognosis? See answer