116540 ⎘
Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2); Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1) Galactosylceramidase (3.2.1.46)
CNS DELIVERY OF THERAPEUTIC AGENTS
#2COMPOSITIONS AND METHODS FOR TREATING METACHROMATIC LEUKODYSTROPHY DISEASE AND RELATED DISORDERS
#3METHODS AND COMPOSITIONS FOR CNS DELIVERY OF ARYLSULFATASE A
#4Lyophilized Pharmaceutical Compositions Comprising A Lipid Nanoparticle
#5COMPOSITIONS AND METHODS OF USING TWO-PROMOTER VECTOR FOR TREATMENT OF LYSOSOMAL STORAGE DISORDERS
#6METHODS AND COMPOSITIONS FOR CNS DELIVERY OF IDURONATE-2-SULFATASE
#7Lyophilized Pharmaceutical Compositions Comprising a Lipid Nanoparticle
#8VLP FOR THE TREATMENT OF LEUKODYSTROPHIES
#9COMPOSITIONS USEFUL IN TREATMENT OF KRABBE DISEASE
#10CNS DELIVERY OF THERAPEUTIC AGENTS
#11Methods and compositions for CNS delivery of iduronate-2-sulfatase
#12Cleavable lipids
#13TREATMENT OF SANFILIPPO SYNDROME TYPE B
#14Methods and compositions for CNS delivery of heparan N-sulfatase
#15COMPOSITIONS USEFUL IN TREATMENT OF KRABBE DISEASE
#16OPTIMIZED GALC GENES AND EXPRESSION CASSETTES AND THEIR USE
#17Methods and compositions for CNS delivery of arylsulfatase A
#18Cleavable lipids
#19CNS delivery of therapeutic agents
#20Methods and compositions for CNS delivery of iduronate-2-sulfatase
#21TREATMENT OF KRABBE DISEASE WITH UMBILICAL CORD BLOOD TRANSPLANTION (UCBT) AND INCREASED GALACTOCEREBROSIDASE (GALC) EXPRESSION
#22Cleavable lipids
#23Methods and compositions for CNS delivery of heparan n-sulfatase
#24PROCESS FOR CONCENTRATION OF A POLYPEPTIDE
#25Cleavable lipids
#26PURIFICATION OF RECOMBINANT HUMAN GALACTOCEREBROSIDE B-GALACTOSIDASE (rhGALC)
#27Therapeutic fusion protein comprising an alpha-n-acetylglucosaminidase and a lysosomal targeting moiety
#28Methods and compositions for CNS delivery of arylsulfatase A
#29Cleavable Lipids
#30CNS delivery of therapeutic agents
#31Combination Therapies for Lysosomal Storage Diseases
#32Process for concentration of a polypeptide
#33PURIFICATION OF RECOMBINANT HUMAN GALACTOCEREBROSIDE B-GALACTOSIDASE (rhGALC)
#34Animal model of Krabbe's disease
#35Cleavable lipids
#36Pharmaceutical composition for treating lysosomal storage disease
#37Treatment of Sanfilippo syndrome type B by intrathecal administration of alpha-n-acetylglucosaminidase
#38Microorganisms genetically engineered to have modified N-glycosylation activity
#39Microorganisms genetically engineered to have modified N-glycosylation activity
#40Process for concentration of a polypeptide
#41METHODS AND COMPOSITIONS FOR CNS DELIVERY OF B-GALACTOCEREBROSIDASE
#42Microorganisms genetically engineered to have modified N-glycosylation activity
#43Glycosylation of molecules
#44PROCESS FOR CONCENTRATION OF A POLYPEPTIDE
#45and HAC1 nucleic acids