Publications by authors named "C Vaillant"
Background: Daridorexant is a dual orexin receptor antagonist approved for the treatment of chronic insomnia disorder.
Aims: Investigate the auditory awakening threshold (AAT), postural stability, and cognitive function during the night following evening administration of daridorexant 25 and 50 mg.
Methods: Double-blind, placebo-controlled, randomized, 3-way (placebo, 25, 50 mg) crossover study in 36 healthy male and female nonelderly adult and elderly subjects (1:1 sex/age ratio).
Dual orexin receptor antagonists (DORAs) are approved for the treatment of sleep onset and/or sleep maintenance insomnia. In the present disclosure, we report the discovery of a new class of DORAs designed to treat sleep disorders requiring a fast onset and a short duration of action (<4 h). We used early human pharmacokinetic-pharmacodynamic (PK-PD) predictions and in vivo experiments to identify DORAs eliciting this specific hypnotic profile.
View Article and Find Full Text PDFArticle Synopsis
- Glutathione synthetase deficiency is a rare genetic disorder caused by mutations in the GSS gene, leading to varying severity levels, from mild hemolytic anemia to severe neurological issues and even neonatal death.
- A study on two fetal siblings revealed multiple congenital anomalies, such as limb malformations, cleft palate, and heart defects, linked to specific genetic variants in the GSS gene.
- Genome sequencing and analysis indicated that these genetic variants likely caused disruptions in protein expression and metabolic processes, suggesting a broader range of phenotypic effects associated with glutathione synthetase deficiency.
Myhre syndrome is a rare genetic disease caused by recurrent gain-of-function variants in SMAD4 (Ile500Thr, Ile500Val, Arg496Cys, and Ile500Met) characterized by postnatal short stature with pseudo-muscular build, joint stiffness, variable intellectual disability, hearing loss, and a distinctive pattern of dysmorphic facial features. The course can be severe in some cases, with life-threatening cardiac and pulmonary complications caused by connective tissue involvement. These progressive features over time make early clinical diagnosis difficult but possible by astute clinicians who evaluate young children with autism or short stature and unusual appearance.
View Article and Find Full Text PDFObjectives: Appraise the evidence for daridorexant 50 mg and 25 mg versus placebo when treating chronic insomnia disorder in terms of number needed to treat (NNT), number needed to harm (NNH), and likelihood to be helped or harmed (LHH).
Methods: NNT, NNH, and LHH were calculated from a 3-month pivotal Phase 3 study ( = 930; randomized 1:1:1 to daridorexant 50 mg, daridorexant 25 mg, or placebo once nightly). Wakefulness after sleep onset, latency to persistent sleep, self-reported total sleep time, Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ), and Insomnia Severity Index were used for the NNT efficacy analysis.