TRPML1 agonists synergize with enzyme replacement therapy in fibroblasts from Pompe disease patients - PubMed
5 hours ago
- #Pompe disease
- #enzyme replacement therapy
- #TRPML1 agonists
- TRPML1 agonists improve M6PR distribution and recycling in Pompe disease fibroblasts.
- Enhanced M6PR availability increases rhGAA uptake and lysosomal trafficking.
- Combination of ERT with TRPML1 agonists boosts GAA activity correction.
- Study supports dual therapy to overcome ERT efficacy limitations.
- Research provides in vitro proof-of-concept for synergistic treatment strategy.