TY - PAT
T1 - COMPOSITIONS AND METHODS FOR ENHANCING N-ACETYLGALACTOSAMINE-6-SULFATASE ENZYME ACTIVITY AND TREATING MUCOPOLYSACCHARIDOSIS TYPE IVA
AU - Alméciga Díaz, Carlos Javier
AU - Cepeda Del Castillo, Jacobo
AU - Guzmán, Esteban
AU - Hidalgo Lanussa, Oscar Alejandro
AU - Olarte Avellaneda, Sergio
AU - Rodriguez López, Edwin Alexander
AU - Zheng, Wei
PY - 2021/12/16
Y1 - 2021/12/16
N2 - The present invention relates to the development of pharmacological chaperones that bind GALNS and compositions thereof. The present invention further relates to the development of methods for treating mucopoiesisaca ridosis type IVA, which comprise administering a therapeutically effective amount of compositions comprising a pharmacological chaperone that binds to GALNS and optionally human recombinant N-acetylgalactosamine-6-sulfatase (hrGALNS). Additionally, the present invention relates to a method for increasing the production of hrGALNS.
AB - The present invention relates to the development of pharmacological chaperones that bind GALNS and compositions thereof. The present invention further relates to the development of methods for treating mucopoiesisaca ridosis type IVA, which comprise administering a therapeutically effective amount of compositions comprising a pharmacological chaperone that binds to GALNS and optionally human recombinant N-acetylgalactosamine-6-sulfatase (hrGALNS). Additionally, the present invention relates to a method for increasing the production of hrGALNS.
UR - https://worldwide.espacenet.com/patent/search/family/073823700/publication/CO2020007242A1?q=CO20200007242
M3 - Invention patent
M1 - WO2021250620
Y2 - 2021/06/10
ER -