Pharmacological chaperone-based therapy for Mucopolysaccharidosis IV A

Project: Research

Project Details

Description

Although ERT with elosulfase alfa is a therapeutic option for mucopolysaccharidosis IVA (MPS IVA) patients, it has limited impact on the bone, immunological issues, and a high cost. In this sense, it is necessary to identify new treatment alternatives for these patients or to improve the therapeutic efficacy of ERT.
StatusFinished
Effective start/end date01/01/21 → 31/07/23

Project funding

  • International
  • NATIONAL MPS SOCIETY