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Genetic Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Genetic Diseases trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Genetic Diseases 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…
This trial will look at a drug called sigvotatug vedotin (SGN-B6A) alone and with pembrolizumab, with or without chemotherapy, to find out whether it is safe fo…
The study seeks primarily to determine whether modulation of systemic and testicular sex steroids balance by aromatase inhibitors will positively affect the met…
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIX…
This is an observational study of children with mild, moderate, or severe hemophilia A who are receiving damoctocog alfa pegol, and are between 7 to \<12 years…
Background: Obesity affects 1 in 5 children in the United States. Childhood obesity often persists into adolescence and adulthood. It can also raise the risk o…