Jesy Nelson, Spinal Muscular Atrophy
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Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular atrophy with respiratory distress type 1 (SMARD1) and type ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with ...
3don MSN
Little Mix alum Jesy Nelson’s 8-month-old twins diagnosed with severe muscle disease, may never walk
Nelson’s twins, Ocean Jade and Story Monroe, have been diagnosed with a rare neuromuscular disorder called Spinal Muscular Atrophy (SMA) Type 1.
Scholar Rock Holding Corporation (NASDAQ:SRRK) President & CEO Jay Backstrom presented at the J.P. Morgan Healthcare Conference. The discussions mainly focused on apitegromab for spinal muscular atrophy (SMA), including its label if approved. They also ...