Search Results 1-10 of 16420 for Spinal muscular atrophy
By comparing the gene expression of motor neurons derived from iPSC cells of phenotypically discordant Spinal Muscular Atrophy (SMA) sibling pairs and ...
Spinal muscular atrophy is a disease that damages nerve cells in the spinal cord called motor neurons. This damage gets worse, and muscles lose strength over ...
Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) · Overview · Participation eligibility · Participating Mayo Clinic ...
The purpose of this study is to identify biomarkers of spinal muscular atrophy in Spinraza-treated patients. Participation eligibility. Participant ...
Spinal muscular atrophy is a rare neuromuscular disorder that is characterized by loss of lower motor neurons and progressive muscle wasting, often leading to ...
Muscular dystrophy is a group of diseases that cause progressive weakness and loss of muscle mass. In muscular dystrophy, abnormal genes (mutations) ...
The main symptoms of the cerebellar type involve poor muscle coordination, known as ataxia. Symptoms may include: Trouble with movement and coordination ...
Risdiplam is used to treat spinal muscular atrophy, a rare nerve and muscle disorder that is characterized by loss of lower motor neurons and progressive ...
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