CORDIS Project
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MeuSIX is developing a gene therapy for Mucopolysaccharidosis VI, a rare genetic disorder. The therapy aims to provide a long-lasting treatment option by delivering a functional gene to patients, potentially improving their quality of life.
Mucopolysaccharidosis VI (MPS VI, or Maroteaux-Lamy syndrome;
OMIM #253200) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase B (ARSB). MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement.
Thus, systemic therapies targeting peripheral organs have the potential to fully correct the MPS VI phenotype.
Enzyme replacement therapy, the current treatment for M…
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
United States, WASHINGTON DC
Type: Company (for-profit)
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
France, EVRY
Type: SME
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: Yes
Italy, Napoli
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Türkiye, Cankaya Ankara
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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