Lipid storage disease Wolman Disease
Conditions
Interventions
None listed
Sponsors
Synageva Biopharma Corp
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - 8 years or older (only adult patients will be included in NL) - No medical condition that would prevent from undergoing MRI or MRS - Ability to understand the full nature and purpose of the study - Confirmation of LAL deficiency
Exclusion criteria
Exclusion criteria: - Contraindications for MRI scanning
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| This is an observational study, no endpoints as such are defined. | — |
Secondary
| Measure | Time frame |
|---|---|
| N A | — |
Countries
Netherlands
Outcome results
None listed