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Treatment Error
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Treatment Error trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Treatment Error trials you may qualify forX-linked hypophosphataemia (XLH) is a rare, hereditary condition. The genetic defect leads to low blood phosphate levels and vitamin D suppression. Phosphate is…
The aims of this clinical trial will test whether or not the onset of nearsightedness is delayed in a group of children randomized to nightly drops in 0.05% atr…
The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standa…
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat…
Arginase 1 deficiency (ARG1-D) is a rare condition in which the body cannot properly break down a substance called arginine. This leads to high levels of argini…
Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, although kidney transplantation may help…
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult p…
Cardiac amyloidosis is a progressive disorder caused by extracellular deposition of amyloid fibrils in the heart, leading to heart failure and impaired cardiac…
The primary objective of this study is to evaluate the safety and tolerability of the dietary supplement, nicotinamide mononucleotide (NMN), in individuals with…