An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
Latest Information Update: 07 Nov 2021
At a glance
- Drugs Vestronidase alfa (Primary)
- Indications Mucopolysaccharidosis VII
- Focus Adverse reactions; First in man; Therapeutic Use
- Sponsors Ultragenyx Pharmaceutical
- 23 Nov 2018 Results published in the Clinical Pharmacokinetics
- 06 Mar 2017 Status changed from active, no longer recruiting to completed.
- 18 May 2016 The trial was completed in Spain.