First-in-human trial aims to tame rare enzyme disorder
Disease control
Recruiting now
This early-stage study tests a new therapy called ISP-001 in 11 people with MPS I (Hurler-Scheie or Scheie types). The main goal is to see if the treatment is safe and tolerable. Participants must be at least 10 years old and able to stay near the study site for a few days after …
Phase: PHASE1 • Sponsor: Immusoft of CA, Inc. • Aim: Disease control
Last updated May 04, 2026 16:20 UTC