Spinal Muscular Atrophy is a group of rare muscle disorders in which the muscles become progressively weakdue to damage to motor neurons.
Check out this story about Faith Fortenberry, a young girl in Texas living with SMA. Her mother reports that in the thirteen months since Faith started on Spinraza, the first and only FDA-approved treatment for SMA, she has experienced "many firsts." Has anyone else tried Spinraza? What was your experience like?
I was wondering is anyone else here diagnosed with more than one condition? I was diagnosed as a child with SMA3 and later as an adult with SMA4. I also have OCA2, Asperger's Syndrome, GERD/IBS, polymyositis, polyarthropathy, FMS, and a variety of other health issues from injuries. I've recovered from 3 different brain aneurysms and more than one bought of cancer though I've been cancer free for over 30 years now.
|Spinal Muscular Atrophy: 4-H Competitor with rare disease inspires others||08/27/2018|
|Doctors said boy wouldn't live past his second birthday. His town just threw a parade for his third||11/24/2019|
|Toronto parents of baby needing $2.8M drug turn to Swiss drugmaker's dose lottery as a last resort||01/01/2020|
|FDA Approves Oral Treatment for Spinal Muscular Atrophy||08/07/2020|
|A race against time: Young boy and his family cope with rare disease||09/12/2020|
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access.
Enrolling is easy.
After these steps, the enrollment process is complete. All other questions are voluntary. However, these questions are important to patients and their families to create awareness as well as to researchers to study rare diseases. This is why we ask our participants to update their information annually or anytime changes to their information occur.
Researchers can contact CoRDS to determine if the registry contains participants with the rare disease they are researching. If the researcher determines there is a sufficient number of participants or data on the rare disease of interest within the registry, the researcher can apply for access. Upon approval from the CoRDS Scientific Advisory Board, CoRDS staff will reach out to participants on behalf of the researcher. It is then up to the participant to determine if they would like to join the study.
Visit sanfordresearch.org/CoRDS to enroll.
:: President of Eminnea Inc., a nonprofit...
I have an 11 year old daughter with Spinal Muscular Atrophy type 2
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