5 Matching Annotations
  1. Last 7 days
  2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Accumulation of membrane repair-associated proteins and mature myostatin are novel markers of muscle pathophysiology in Pompe disease
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    pmc.ncbi.nlm.nih.gov/articles/PMC13548533/
  3. Apr 2026
  4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Longitudinal characterization of Gaac.1826dupA mice reveals the cardiac, myopathic and biochemical phenotypes of Pompe disease
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    pmc.ncbi.nlm.nih.gov/articles/PMC13035063/
  5. Mar 2025
  6. link.springer.com link.springer.com
    Rat models of musculoskeletal lysosomal storage disorders and their role in pre-clinical evaluation of gene therapy approaches
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    link.springer.com/article/10.1007/s00335-025-10121-3
  7. Oct 2020
  8. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov
    Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
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    pubmed.ncbi.nlm.nih.gov/33039711/
  9. Jan 2020
  10. elifesciences.org elifesciences.org
    Impaired lysosomal acidification triggers iron deficiency and inflammation in vivo
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    elifesciences.org/articles/51031