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Spinal Muscular Atrophy

What is Spinal Muscular Atrophy?

Spinal Muscular Atrophy is a group of rare muscle disorders in which the muscles become progressively weakdue to damage to motor neurons.

 

Spinal Muscular Atrophy is a group of rare muscle disorders in which the muscles become progressively weakdue to damage to motor neurons.
Acknowledgement of Spinal Muscular Atrophy has not been added yet.
3.0http://www.orpha.net
Synonyms for Spinal Muscular Atrophy has not been added yet.
Cause of Spinal Muscular Atrophy has not been added yet.
Symptoms for Spinal Muscular Atrophy has not been added yet.
Diagnosis of Spinal Muscular Atrophy has not been added yet.
Diagnostic tests of Spinal Muscular Atrophy has not been added yet
Treatments of Spinal Muscular Atrophy has not been added yet.
Prognosis of Spinal Muscular Atrophy has not been added yet.
Tips or Suggestions of Spinal Muscular Atrophy has not been added yet.
References of Spinal Muscular Atrophy has not been added yet.
See Disorder Resources for news link on FDA approved therapy for Spinal Muscular Atrophy Created by shannon.ashoori
Last updated 7 Aug 2020, 08:30 PM

Posted by shannon.ashoori
7 Aug 2020, 08:30 PM

Spinraza for SMA Created by RareshareTeam
Last updated 29 Jul 2018, 07:52 PM

Posted by RareshareTeam
29 Jul 2018, 07:52 PM

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?

Hi Everyone Created by Nimonic
Last updated 10 Sep 2016, 10:05 PM

Posted by Nimonic
10 Sep 2016, 10:05 PM

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.

Community External News Link
Title Date Link
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
Woman with rare muscular disease sings to exercise her lungs. Her songs inspire others to chase their dreams 05/02/2021
Child fights for her life by taking the 'most expensive drug in the world' 05/21/2021
Columbus mom lost one daughter to a rare disease. A new drug is helping another 06/12/2021
New treatment for a rare genetic disease to be made available on NHS 11/20/2021
Parents Lose Two Babies Weeks Apart as Child Dies of Rare Genetic Disease 02/06/2022
Aspen’s story: A 4-year-old living with a rare disease 01/07/2023
I suffer from the world's most beautiful disease - and also the most expensive to treat 06/18/2023
Hailey boy discusses his bout with a rare disease 02/17/2024
Community Resources
Title Description Date Link

Clinical Trials


Cords registry

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.

  1. Complete the screening form.
  2. Review the informed consent.
  3. Answer the permission and data sharing questions.

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.

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Community User List

I'm from a large family. I have several half siblings, step siblings, and one full sibling. Several of us have AIS. But a few have other conditions. Some are non affected carriers or non carriers....
My daughter has a 3 month old baby girl that the Dr's are 99%positive has SMA with respiratory distress and I am trying to find out as much as I can about this very rare disease.
i have sma 3 and just want soomeone to talk to . i recently lost my mother and am very depressed .but otherwise i love shopping, fishing,and making jewelry.
:: Creator of SMASpace, an international Spinal Muscular Atrophy (SMA) community (www.smaspace.com), and father to kids with Spinal Muscular Atrophy.

 

:: President of Eminnea Inc., a nonprofit...
My daughter, Gwendolyn, was born perfectly healthy in October 2007 and diagnosed with the Type 1 Spinal Muscular Atrophy (SMA1) at 6 months.

 

 

http://www.PetitionToCureSMA.com
I live in Toronto,Ontario. I am the president of the Fightsma chapter in Canada.

 

 

I have an 11 year old daughter with Spinal Muscular Atrophy type 2

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Discussion Forum

Spinraza for SMA

Created by RareshareTeam | Last updated 29 Jul 2018, 07:52 PM

Hi Everyone

Created by Nimonic | Last updated 10 Sep 2016, 10:05 PM


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