Metabolic

Mucopolysaccharidosis

Also known as MPS I (Hurler), MPS II (Hunter), MPS III (Sanfilippo), MPS IV (Morquio)

ORPHA:79213 ↗Gene Multiple (IDUA Gene IDS Gene SGSH Gene GALNS Gene ARSB...)Prevalence 1-9 per 1,000,000 (Orphanet, Type 1)Onset Childhood (type-dependent)Genetic (autosomal recessive or X-linked)

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Patient organisations

National MPS SocietyPatient association
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Registry: MPS Society Patient Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.

NORD ↗GARD ↗Orphanet ORPHA:79213 ↗

About Mucopolysaccharidosis

Mucopolysaccharidoses are a group of metabolic disorders caused by enzyme deficiencies that prevent the body from breaking down glycosaminoglycans (formerly called mucopolysaccharides). These complex sugars accumulate in cells throughout the body, causing progressive damage to organs, bones, and the nervous system. Each MPS type is caused by a different enzyme deficiency and has its own clinical course.

Common clinical features

Joint stiffnessScoliosisCorneal opacitySplenomegalyCoarse facial featuresDevelopmental regressionRecurrent upper respiratory tract infectionsAbnormal heart morphology

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

4 approved treatments and 9 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Vestronidase Alfa (Mepsevii)Approved: Galsulfase (Aryplase)Approved: Laronidase (Aldurazyme)Approved: Elosulfase Alfa (Bmn 110, rhgalns, n-acetylgalactosamine-6-sulfatase, chondroitinsulfatase, galactose-6-sulfate sulfatase, ec=3.1.6.4)

Phase 2/3Idursulfase (Elaprase)

Phase 2/3Somatropin (Genotropin)

Phase 1/2Lesinidase Alfa

Phase 1/2Valanafusp Alfa

Phase 1/2Clemidsogene Lanparvovec

Phase 1/2Cyclosporine (Capimune)

Phase 1/2Rebisufligene Etisparvovec

Phase 1/2Azathioprine (Azamune)

+ 1 more in development

Before you apply

Things trial teams commonly ask about for Mucopolysaccharidosis. Not eligibility rules; those are set by each study.

MPS type (I through VII) must be confirmed by enzyme assay or genetic testing before applying to trials
CNS involvement (present in MPS I, II, III, VII) determines whether intrathecal delivery trials apply
ERT experience and anti-drug antibody levels are often required at enrollment

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).