X
Search Filters
Format Format
Subjects Subjects
Subjects Subjects
X
Sort by Item Count (A-Z)
Filter by Count
laronidase (109) 109
humans (103) 103
enzyme replacement therapy (95) 95
index medicus (95) 95
mucopolysaccharidosis (72) 72
enzymes (69) 69
male (59) 59
genetics & heredity (58) 58
female (57) 57
pediatrics (49) 49
enzyme-replacement therapy (48) 48
child (44) 44
medicine, research & experimental (44) 44
child, preschool (43) 43
l-iduronidase laronidase (42) 42
children (39) 39
iduronidase - therapeutic use (39) 39
mucopolysaccharidosis i - drug therapy (39) 39
glycosaminoglycans (34) 34
infant (34) 34
adolescent (33) 33
health aspects (33) 33
disease (32) 32
care and treatment (31) 31
mucopolysaccharidosis i - therapy (31) 31
treatment outcome (30) 30
adult (28) 28
hurler-syndrome (28) 28
bone-marrow-transplantation (27) 27
transplantation (27) 27
follow-up (24) 24
mucopolysaccharidosis type i (24) 24
mucopolysaccharidosis i - diagnosis (23) 23
alpha-l-iduronidase (22) 22
stem-cell transplantation (22) 22
animals (21) 21
mps-i (21) 21
mucopolysaccharidosis i (21) 21
endocrinology & metabolism (20) 20
young adult (20) 20
biopharmaceutics (19) 19
enzyme replacement therapy - methods (19) 19
genetics (19) 19
hurler syndrome (19) 19
glycosaminoglycans - urine (18) 18
hematopoietic stem cell transplantation (18) 18
medicine (18) 18
diagnosis (17) 17
hematopoietic stem cells (17) 17
research (17) 17
antibodies (16) 16
biochemistry & molecular biology (16) 16
congenital, hereditary, and neonatal diseases and abnormalities (16) 16
lysosomal storage disorders (16) 16
analysis (15) 15
follow-up studies (15) 15
mucopolysaccharidosis i - pathology (15) 15
mucopolysaccharidosis-i (15) 15
nutritional and metabolic diseases (15) 15
phenotype (15) 15
mucopolysaccharidoses (14) 14
pharmacology & pharmacy (14) 14
recombinant proteins - therapeutic use (14) 14
safety (14) 14
efficacy (13) 13
hurler (13) 13
iduronidase - administration & dosage (13) 13
medicine & public health (13) 13
mucopolysaccharidosis i - genetics (13) 13
mucopolysaccharidosis type-i (13) 13
patients (13) 13
stem cells (13) 13
abridged index medicus (12) 12
iduronidase - adverse effects (12) 12
infant, newborn (12) 12
mps i (12) 12
mucopolysaccharidosis i - complications (12) 12
pharmacology (12) 12
experience (11) 11
iduronidase - genetics (11) 11
internal medicine (11) 11
mice (11) 11
mucopolysaccharidosis i - physiopathology (11) 11
n-acetylgalactosamine 4-sulfatase (11) 11
glycosaminoglycans - metabolism (10) 10
hormone therapy (10) 10
idursulfase (10) 10
management (10) 10
medical genetics (10) 10
metabolic disorders (10) 10
age (9) 9
combined modality therapy (9) 9
fabry-disease (9) 9
ii hunter-syndrome (9) 9
immunology (9) 9
long-term (9) 9
lysosomal storage diseases (9) 9
medical research (9) 9
middle aged (9) 9
mucopolysaccharidoses - complications (9) 9
more...
Language Language
Language Language
X
Sort by Item Count (A-Z)
Filter by Count
English (143) 143
French (5) 5
Portuguese (2) 2
German (1) 1
Japanese (1) 1
Polish (1) 1
Spanish (1) 1
more...
Publication Date Publication Date
Click on a bar to filter by decade
Slide to change publication date range


Pediatrics & Neonatology, ISSN 1875-9572, 10/2019, Volume 60, Issue 5, pp. 577 - 578
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2014, Volume 111, Issue 2, pp. 63 - 72
The mucopolysaccharidoses (MPS), a group of rare genetic disorders caused by defects in glycosaminoglycan (GAG) catabolism, are progressive, multi-systemic... 
Pre-symptomatic | Mucopolysaccharidosis | Laronidase | Enzyme replacement therapy | Galsulfase | Idursulfase | MEDICINE, RESEARCH & EXPERIMENTAL | HURLER-SYNDROME | JOINT DISEASE | FOLLOW-UP | N-ACETYLGALACTOSAMINE 4-SULFATASE | LYSOSOMAL STORAGE DISORDERS | MPS-I | HUNTER OUTCOME SURVEY | ENDOCRINOLOGY & METABOLISM | GENETICS & HEREDITY | L-IDURONIDASE LARONIDASE | HEPARAN-SULFATE | STEM-CELL TRANSPLANTATION | N-Acetylgalactosamine-4-Sulfatase - therapeutic use | Recombinant Proteins - therapeutic use | Humans | Child, Preschool | Secondary Prevention | Dysostoses - physiopathology | Joints - physiopathology | Respiratory System - enzymology | Heart Valves - drug effects | Mucopolysaccharidoses - complications | Mucopolysaccharidoses - drug therapy | Enzyme Replacement Therapy | Heart Valves - enzymology | Dysostoses - enzymology | Joints - enzymology | Mucopolysaccharidoses - physiopathology | Glycosaminoglycans - metabolism | Joints - drug effects | Mucopolysaccharidoses - enzymology | Dysostoses - complications | Clinical Trials as Topic | Dysostoses - drug therapy | Respiratory System - drug effects | Respiratory System - physiopathology | Iduronate Sulfatase - therapeutic use | Heart Valves - physiopathology | Enzymes | Care and treatment | Glycosaminoglycans | Physiological aspects | Health aspects | Biopharmaceutics
Journal Article
Value in Health Regional Issues, ISSN 2212-1099, 05/2019, Volume 18, pp. 165 - 169
Mucopolysaccharidosis I (MPS-I) is one of the most common types of MPS and lysosomal storage diseases, which impose considerable amount of economic burden on... 
laronidase | Iran | resource utilization | MPS-I | healthcare
Journal Article
Pediatrics, ISSN 0031-4005, 01/2009, Volume 123, Issue 1, pp. 229 - 240
Journal Article
Cochrane Database of Systematic Reviews, ISSN 1469-493X, 09/2013, Volume 2013, Issue 9
Background Mucopolysaccharidosis type I can be classified as three clinical sub-types; Hurler syndrome, Hurler-Scheie syndrome and Scheie syndrome, with the... 
MEDICINE, GENERAL & INTERNAL | EFFICACY | SAFETY | DISEASE | L-IDURONIDASE LARONIDASE | GAUCHER REGISTRY | EXPERIENCE | STEM-CELL TRANSPLANTATION
Journal Article
Journal Article
Journal of Inherited Metabolic Disease, ISSN 0141-8955, 09/2019, Volume 42, Issue 5, pp. 762 - 775
Orphan medicinal products (OMPs) are often authorized based on pivotal phase II and III trials that do not always meet high quality criteria. Laronidase is an... 
effectiveness | enzyme replacement therapy | laronidase | orphan drug | mucopolysaccharidosis type I | MEDICINE, RESEARCH & EXPERIMENTAL | PHENOTYPE | FOLLOW-UP | EFFICACY-EFFECTIVENESS GAP | NATURAL-HISTORY | MPS-I | MOTION | ENDOCRINOLOGY & METABOLISM | GENETICS & HEREDITY | RANGE
Journal Article
Genetics in Medicine, ISSN 1098-3600, 09/2017, Volume 19, Issue 9, pp. 975 - 982
The US Secretary of Health and Human Services recommended in February 2016 that mucopolysaccharidosis type 1 (MPS I) be added to the recommended uniform... 
evidence-based practice | dried bloodspot testing | public health | mucopolysaccharidosis type I | HURLER-SYNDROME | HEMATOPOIETIC-CELL TRANSPLANTATION | LARONIDASE | BONE-MARROW-TRANSPLANTATION | GUIDELINES | ATTENUATED MPS I | DISORDERS | CHILDREN | PERSPECTIVES | GENETICS & HEREDITY | ENZYME REPLACEMENT THERAPY | Medical screening | Cognitive ability
Journal Article