Hyperargininemia Deficiency of an enzyme called arginase 1
Conditions
Interventions
Loargys is a medicine designed to help manage arginase 1 deficiency in patients aged 2 years and older. It works by lowering levels of a substance called arginine in the blood. The treatment has been
Sponsors
Immedica Pharma AB
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Enrolled in the main E-IMD registry2. Diagnosis of ARG1-D3. Written informed consent for use of E-IMD registry data in accordance with this study protocol.
Exclusion criteria
Exclusion criteria: There are no exclusion criteria for the PAES in addition to the exclusion criteria in the E-IMD registry protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the durability of treatment response in patients with arginase 1 deficiency treated with Loargys in standard clinical care. | — |
Secondary
| Measure | Time frame |
|---|---|
| -To assess clinical outcomes in patients with arginase 1 deficiency treated with Loargys in standard clinical care.-To describe treatment in patients with arginase 1 deficiency treated with Loargys in standard clinical care. | — |
Countries
Austria, France, Germany, Netherlands, Portugal
Contacts
Public ContactMR Rudebeck
Immedica Pharma AB
Outcome results
None listed