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Inherited Metabolic Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Metabolic Diseases trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Metabolic Diseases trials you may qualify forThe purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standa…
Background: Fanconi anemia (FA) is a rare, inherited cancer syndrome. FA causes a range of physical issues. Children with FA may have abnormal features; these…
The U-TRANS study was initiated by Gustave Roussy, which is its sponsor\*. It is part of the Interception Program and aims to reduce the consumption of ultra-pr…
Cardiovascular disease is the leading cause of death worldwide. Advanced cardiovascular imaging using Magnetic Resonance Imaging (MRI) has proven to be effectiv…
The goal of this observational study is to develop a registry of Brazilian patients with hereditary cardiovascular diseases, combining clinical and genomic data…
Chediak-Higashi syndrome (CHS) is a rare autosomal recessive disorder characterized in its classical form by oculocutaneous albinism, a bleeding diathesis, recu…
Fabry disease is a rare, inherited condition that can affect multiple organs, including the heart, kidneys, and nervous system. Many people with Fabry disease l…
To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunli…
The purpose of this study is to evaluate the safety and efficacy of multiple doses of PLX-200 in patients with CLN3 disease.