Mucopolysaccharidosis (MPS) Treatment Market Witnesses Research and Development of New Therapies
Mucopolysaccharidosis refers to a group of inherited conditions that are characterized by the inability of the body to properly breakdown mucopolysaccharides (long chains of sugar molecules that are found throughout the body). Seven distinct clinical types and numerous subtypes of the mucopolysaccharidoses have been identified. Hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy with a recombinant form of human IDUA administered intravenously are current disease modifying therapies for MPS. The mucopolysaccharidosis (MPS) treatment market is witnessing R&D of new therapies. For instance, in February 2021, Orchard Therapeutics, a gene therapy company, announced new data from several of its hematopoietic stem cell (HSC) gene therapies in development for neurodegenerative disorders at the 17th Annual WORLDSymposium. The presentation included interim data on multiple clinical outcomes for OTL-203 in MPS-I, encouraging preliminary biomarker data for OTL-201...