What’s Up Doc? New Therapies For Spinal Muscular Atrophy Patients

Q: Is there a new treatment for spinal muscular atrophy?A: Spinal muscular atrophy (SMA) is a group of recessively inherited disorders (the patient must inherit a defective gene from both of their parents) that manifest as muscle weakness (for example affecting the patient’s ability to walk, and potentially affecting their ability to breath), occurring in up to one in every 10,000 people.

Topics:  spinal   sma   in   patient   ability   
BING NEWS:
  • Family raises funds for son’s spinal muscular atrophy treatment
    Luis's wheelchair is part of his everyday life with spinal muscular atrophy and he faces challenges with mobility and living every day.
    11/29/2024 - 8:06 am | View Link
  • SMA infants show loss of spinal motor, sensory neurons: Study
    Children with severe SMA have substantial loss of motor and sensory neurons in the thoracic region of the spinal cord, a study showed.
    11/29/2024 - 12:17 am | View Link
  • Grant supports Revir’s work in spinal and bulbar muscular atrophy
    The collaboration aims to identify a small molecule that targets mutant androgen receptor (AR) mRNA splicing and causes selective destruction of the disease-causing mRNA.
    11/27/2024 - 11:00 am | View Link
  • Biohaven's spinal muscular atrophy drug fails to meet study goal
    By Mariam Sunny (Reuters) -Biohaven said on Monday its experimental treatment for a rare neuromuscular disorder failed to significantly improve motor function in a late-stage trial, sending the shares ...
    11/25/2024 - 6:06 am | View Link
  • Biohaven's Spinal Muscular Atrophy Treatment Fails Primary Goal in Study; Plans Treatment for Obesity
    The New Haven, Conn., biopharmaceutical company said Monday that taldefgrobep had shown clinically meaningful improvements in motor function in spinal muscular atrophy patients, but the treatment arm ...
    11/25/2024 - 12:33 am | View Link
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