Search Results 1-10 of 17251 for Spinal muscular atrophy

  1. Spinal Muscular Atrophy Clinical Trials - Mayo Clinic Research

    By comparing the gene expression of motor neurons derived from iPSC cells of phenotypically discordant Spinal Muscular Atrophy (SMA) sibling pairs and ...

  2. Spinal Muscular Atrophy (SMA) Clinical Trials - Mayo Clinic Research

    Spinal muscular atrophy (SMA) ... The purpose of this study is to identify biomarkers of spinal muscular atrophy in Spinraza-treated patients. ... The purpose of ...

  3. A Study to Evaluate Pluripotent Stem Cell Models for Spinal Muscular Atrophy (SMA) Patients - Mayo Clinic

    By comparing the gene expression of motor neurons derived from iPSC cells of phenotypically discordant Spinal Muscular Atrophy (SMA) sibling pairs and ...

  4. A Study to Identify Biomarkers of Spinal Muscular Atrophy in Spinraza-Treated Patients - Mayo Clinic

    The purpose of this study is to identify biomarkers of spinal muscular atrophy in Spinraza-treated patients. Participation eligibility. Participant ...

  5. Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) - Mayo Clinic

    Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) · Overview · Participation eligibility · Participating Mayo Clinic ...

  6. Onasemnogene abeparvovec-xioi (intravenous route) - Side effects & uses - Mayo Clinic

    Spinal muscular atrophy is a rare neuromuscular disorder that is characterized by loss of lower motor neurons and progressive muscle wasting, often leading to ...

  7. Sharing Mayo Clinic: Every day you don't lose, you are winning

    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 ...

  8. Onasemnogene abeparvovec-brve (intrathecal route) - Side effects & uses - Mayo Clinic

    Spinal muscular atrophy is a rare neuromuscular disorder that is characterized by loss of lower motor neurons and progressive muscle wasting, often leading to ...

  9. Neuromuscular Diseases Clinical Trials - Mayo Clinic Research

    ... Spinal Muscular Atrophy (SMA) sibling pairs and individual cases of SMA, we hope to identify potential protective gene modifiers of SMA. Identifying ...

  10. Multiple system atrophy - Symptoms and causes - Mayo Clinic

    The main symptoms of the cerebellar type involve poor muscle coordination, known as ataxia. Symptoms may include: Trouble with movement and coordination ...