Why it matters: Modeling Pompe skeletal muscle metabolism defects in vitro is a challenge. Thanks to the sensitive imaging readouts developed, early and late Pompe disease models showed distinct cellular phenotypes.
Indeed, there are very few reports using primary human Pompe myotubes and even fewer comparing EOPD and LOPD myotubes side-by-side. Having both Pompe models available enables a more comprehensive evaluation of new therapeutics targeting muscle defects in Pompe patients.
Our solution: Using patient-derived cells on our MyoScreen™ platform, we’ve created physiologically relevant models that:
✅ Replicate essential pathological features
✅ Enable quantitative, functional assessments
✅ Support HT/HC drug screening
✅ Bridge the gap from bench to bedside
Our expertise in disease modeling includes:
- Neuromuscular diseases
- Other genetic disorders
- Metabolic disorders (muscle weakness and waste, obesity, diabetes)
Everything with MyoScreen™ platform is tailored to your needs through a flexible R&D partnership model.
📄 Read the full abstract from WMS2024 in Neuromuscular Disorders journal
For more information, please contact us !
