Mucopolysaccharidosis type 1

MONDO:0001586

The most common type of mucopolysaccharidosis. It is inherited in an autosomal recessive pattern. It comprises a group of lysosomal storage diseases which includes the most severe form (Hurler syndrome) and the mildest form (Scheie syndrome).

Also known as: Alpha-L-iduronidase deficiency, MPS1, MPSI, Mucopolysaccharidosis Type I, lipochondrodystrophy, mucopolysaccharidosis type 1, mucopolysaccharidosis type I, Hurler syndrome

19 clinical trials for this condition and its sub-types, 11 tagged with Mucopolysaccharidosis type 1 itself.

Follow this condition to get notified about new trials

Where it sits in the disease tree

Browse by category →

Part of

↑ Bone disorder (6290) ↑ Eye disorder (3068) ↑ Mucopolysaccharidosis (61)

Sub-types of Mucopolysaccharidosis type 1

Including sub-types (19) Tagged with Mucopolysaccharidosis type 1 (11)

Trials to join now! 5 Not yet recruiting 1 Not yet finished but already full! 10 Completed 2 Terminated 1

Gene Editing's lasting impact: a 10-Year safety watch

Knowledge-focused Stopped early

This study checks on people who previously received gene editing for hemophilia B or mucopolysaccharidosis (MPS) I or II. No new treatment is given; instead, participants are monitored for up to 10 years to see if any new health problems or worsening of existing conditions appear…

Sponsor: Sangamo Therapeutics • Aim: Knowledge-focused

Last updated Aug 15, 2026 00:00 UTC

Mucopolysaccharidosis type 1

Where it sits in the disease tree

Gene Editing's lasting impact: a 10-Year safety watch