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A large clinical trial with ganaxolone is currently ongoing in children and adolescents with fragile X syndrome between 6 to 17 years of age (Wijetunge et al., 2013

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In A Study of Olezarsen Administered to Patients With Familial Chylomicronemia Syndrome (BALANCE), 66 patients with genetically confirmed FCS (homozygous or heterozygous compound loss function mutations in the genes for LPL, apoC2, apoA5, LMF-1 , or GPIHBP1 ) and fasting TG 880 mg/dl ( 10 mmol/l) were randomized to receive placebo or olezarsen at 50 or 80 mg monthly [106]

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Oncotarget 8 (57), 9663696648 (2017)