“Phase III, Single-arm, Open-label, International, Multi-centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients with Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid-lowering Therapy”; Studio clinico - Coinvestigatrice
Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) randomized multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-16); Studio clinico - Coinvestigatrice
A Multicenter, Open-label, Phase 2, Extension Trial to Study the Long-term - Coinvestigatrice
Safety in Participants With PROS or Proteus Syndrome Who Are Currently Being Treated with Miransertib in Other Studies (MK-7075) - Coinvestigatrice
LIPIGEN (LIPID TRANSPORT DISORDERS ITALIAN GENETIC NETWORK): Registro delle dislipidemie familiari in Italia
EPIK-P2: A Phase II double-blind study with an upfront, 16-week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS); Studio clinico - Coinvestigatrice
“A Phase 1/2 Study of ARQ 092 in Patients with Overgrowth Diseases and Vascular Anomalies with Genetic Alterations of the PI3K/AKT Pathway”; Studio clinico - Coinvestigatrice
“Double-blind, Randomized, Multicenter, Placebo-controlled, Parallel Group Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for Low Density Lipoprotein-cholesterol (LDL-C) Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH)”; Studio clinico - Coinvestigatrice
“A Randomized, Double-Blind, Placebo-Controlled Study Followed by an Open Label Treatment Period to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Heterozygous Familial Hypercholesterolemia”; Studio clinico - Coinvestigatrice
“STAGE (STAtin and GEnes): Studio osservazionale dei determinanti clinici, biochimici, di farmacocinetica e farmacogenetica per la risposta alla terapia ipocolesterolemizzante con atorvastatina nei bambini affetti da Ipercolesterolemia Familiare in eterozigosi; Studio clinico - Coinvestigatrice
