Aerosolized lancovutide in adolescents (≥12 years) and adults with cystic fibrosis - a randomized trial.

Background: Lancovutide activates a chloride channel (TMEM-16A) other than the cystic fibrosis (CF) transmembrane conductance regulator protein and could benefit CF patients.

Methods: In this randomized, multi-center, double-blind, placebo-controlled, parallel-group trial 161 patients ≥12 years with a confirmed diagnosis of CF were randomized to either placebo (saline) or active drug in 3 different dosing schemes of 2.5mg inhaled lancovutide (once daily, every other day or twice a week) for eight weeks.

Phenotype of children with inconclusive cystic fibrosis diagnosis after newborn screening.

Objective: To characterize the phenotypic expression of children with conductance regulator-related metabolic syndrome (CRMS)/cystic fibrosis screen positive inconclusive diagnosis (CFSPID) designation after positive newborn screening, reassign labeling if applicable and better define these children's prognosis.

Methods: A multicenter cohort with CRMS/CFSPID designation was matched with cystic fibrosis (CF)-diagnosed cohort. Cohorts were prospectively compared on baseline characteristics, cumulative data and when they reached 6 to 7 years at endpoint assessment.

Gait abnormalities in people with Dravet syndrome: A cross-sectional multi-center study.

Objective: To quantify gait abnormalities in people with Dravet syndrome (DS).

Methods: Individuals with a confirmed diagnosis of DS were enrolled, and stratified according to knee flexion at initial contact (IC) and range of motion (ROM) during stance (atypical crouch: knee flexion >20° at IC and knee ROM >15° during stance; straight: knee flexion <20° at IC). A 1D ANOVA (α = 0.

Intra-articular botulinum toxin injection in complex regional pain syndrome: Case report and review of the literature.

Complex regional pain syndrome (CRPS) is characterized by hyperalgesia, autonomic and trophic alterations of bones, muscles and skin. It is supported by neurogenic inflammation and impairment of sympathetic nervous system. Botulinum Toxin (BTX) is an option for the management of pain, with level B evidence of efficacy in neuropathic, joint and myofascial pain syndrome.

LRP1 expression in colon cancer predicts clinical outcome.

Unlabelled: LRP1 (low-density lipoprotein receptor-related protein 1), a multifunctional endocytic receptor, has recently been identified as a hub within a biomarker network for multi-cancer clinical outcome prediction. As its role in colon cancer has not yet been characterized, we here investigate the relationship between LRP1 and outcome.

Materials And Methods: mRNA expression was determined in colon adenocarcinoma and paired colon mucosa samples, as well as in stromal and tumor cells obtained after laser capture microdissection.