Does Spinal muscular atrophy with respiratory distress type 1 have a cure?
Is there a cure for Spinal muscular atrophy with respiratory distress type 1? Current treatment landscape and research progress, medically reviewed, plus patient experiences.
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Kate started showing symptoms at 6 weeks old and passed away at 12.5 weeks old. Her official SMARD1 diagnosis wasn't received until 2 weeks after her death. her full story can be found here:
http://karryonkate.blogspot.com/p/about-kate.html?m=...
Truett is technically undiagnosed, although some doctors at Childrens Hospital of Colorado and Johns Hopkins Hospital in Baltimore believe he has a unknown version of SMARD. On the SMARD gene, he has one variance and one "normal" spelling.
Truett h...