mucopolysaccharidoses

Are there any ongoing research or future prospects?

Research into MPS is ongoing, with several promising avenues being explored:
- Gene therapy aims to correct the underlying genetic defect by delivering a functional copy of the gene to the patient's cells.
- Substrate reduction therapy focuses on reducing the production of GAGs to decrease their accumulation.
- New enzyme replacement therapies and delivery methods are being developed to improve efficacy and reduce side effects.

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