3 results
Approved WMORecruiting
Main objectives: 1. to investigate the prevalence of vision abnormalities in 9- and 10 year old children; 2. to investigate the percentage of these vision abnormalities that were detected the VOV screening programme;3. to investigate whether these…
Approved WMORecruiting
Objective Endpoint• To assess the clinical safety and tolerability of leniolisib in pediatric patients (aged 4 to 11 years) with APDS• Incidence of treatment-emergent AEs (TEAEs), SAEs, and AEs leading to discontinuation of study drug• Change from…
Approved WMORecruiting
The objective is to determine whether etidronate can halt or attenuate disease progression in patients with Fahr*s disease or syndrome.