ایدرونیداز
Sign in to saveAlso known as mucopolysaccharidosis type I, IDUA, α-L-iduronidase, alpha-L-iduronidase, human alpha-L-iduronidase, alpha-L-iduronidase precursor
Iduronidase (, L-iduronidase, α-L-iduronidase, laronidase), sold as Aldurazyme, is an enzyme with the systematic name glycosaminoglycan α-L-iduronohydrolase. It catalyses the hydrolysis of unsulfated α-L-iduronosidic linkages in dermatan sulfate.
Key facts
- Drug.P
- 4
- Drug.DailyMedID
- Laronidase
- Drug.O
- 1261
- Drug.S
- 12
- Drug.pregnancy_AU
- B2
- Protein.Name
- iduronidase, α-L-
- Protein.HGNCid
- 5391
- Protein.Symbol
- IDUA
- Protein.EntrezGene
- 3425
- Protein.OMIM
- 252800
- Protein.RefSeq
- NM_000203
- Protein.UniProt
- P35475
- Protein.ECnumber
- 3.2.1.76
- Protein.Chromosome
- 4
- Protein.Arm
- p
- Protein.Band
- 16.3
- Drug.USAN
- Laronidase
- Drug.JAN
- Laronidase (genetical recombination)
via Wikipedia infobox
Chemical data
Drug data · ChEMBL
- Max clinical phase
- Approved
- Molecule type
- Enzyme
- First approval
- 2003
- Admin. routes
- parenteral
- Indications
- Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome, mucopolysaccharidosis
via ChEMBL · EBI
Protein · UniProt
Alpha-L-iduronidase
- Gene
- IDUA
- Organism
- Homo sapiens (Human)
- Length
- 653 aa
- Molecular mass
- 72,670 Da
- Evidence
- 1: Evidence at protein level
Lysosomal hydrolase responsible for the degradation of the glycosaminoglycans heparan sulfate and dermatan sulfate
3D-structureAlternative splicingDisease variantDisulfide bondGlycoproteinGlycosidaseHydrolaseLysosome
View on UniProt →
Swiss-Prot (reviewed) · via UniProt
Research
134 papers- Laronidase.2006
- Laronidase.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2002
- Enzyme replacement therapy with laronidase (Aldurazyme(®)) for treating mucopolysaccharidosis type I.The Cochrane database of systematic reviews · 2019
- Laronidase treatment of mucopolysaccharidosis I.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2005
- Laronidase for treating mucopolysaccharidosis type I.Genetics and molecular research : GMR · 2007
via PubMed
Clinical Trials
18 registered- ACTIVE_NOT_RECRUITINGEvaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.Masonic Cancer Center, University of Minnesota · NCT05634512
- PHASE1TERMINATEDIntrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) IPatricia I. Dickson, M.D. · NCT00215527
- PHASE3COMPLETEDStudy of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) DiseaseGenzyme, a Sanofi Company · NCT00258011
- PHASE4COMPLETEDChina Post-marketing Surveillance (PMS) Study of Aldurazyme®Genzyme, a Sanofi Company · NCT05134571
- COMPLETEDA Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care SettingSanofi · NCT05073783
- TERMINATEDLaronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler SyndromeMasonic Cancer Center, University of Minnesota · NCT01572636
Wikidata facts
- Instance of
- protein
- Has use
- medication
Show 6 more facts
- medical condition treated
- mucopolysaccharidosis I
- has characteristic
- biopharmaceutical
- legal status (medicine)
- boxed warning
- found in taxon
- Homo sapiens
- cell component
- extracellular exosome
- exact match
- purl.uniprot.org/uniprot/P35475
via Wikidata · CC0