A Bay Area pharmaceutical firm has announced positive data from a phase 3 clinical trial evaluating Chenodal tablets to treat adults with cerebrotendinous xanthomatosis, an autosomal, recessive, progressive genetic disorder.
According to a release from Mirum Pharmaceuticals Inc., the randomized, withdrawal, placebo-controlled study, called RESTORE, evaluated the safety and efficacy of Chenodal (chenodiol) by measuring the level of urine bile alcohols.
Study protocol involved a 4-week screening period, a treatment period of 6 months and a follow-up phone call 30 days after administration of