Proteostasis Therapeutics Appoints David Arkowitz to Board of Directors
BOSTON, March 7, 2019 -- (Healthcare Sales &Marketing Network) -- Proteostasis Therapeutics, Inc. (NASDAQ: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (C... Biopharmaceuticals, Personnel Proteostasis Therapeutics, transmembrane conductance regulator, cystic fibrosis
Improved lung function, increased BMI, reduced need for IV antibiotics seen after one year of therapy
Authors: Lee L, Smith-Whitley K, Banks S, Puckrein G Abstract Sickle cell disease (SCD) is an inherited blood disorder most common among African American and Hispanic American persons. The disease can cause substantial, long-term, and costly health problems, including infections, stroke, and kidney failure, many of which can reduce life expectancy. Disparities in receiving health care among African Americans and other racial/ethnic minority groups in the United States are well known and directly related to poor outcomes associated with SCD. As an orphan disease-one that affects
FRIDAY, Oct. 11, 2019 -- After one year of therapy with lumacaftor-ivacaftor in a real-world setting, adolescents and adults with cystic fibrosis and Phe508del homozygous mutation experienced an improvement in lung function, increase in body mass...
ABSTRACT Objective: To evaluate the association of body mass index (BMI) and albumin with pulmonary function in cystic fibrosis (CF) pediatric subjects. Methods: This is a cross-sectional study with clinically stable CF ’ s subjects. Clinical (pulmonary function) and nutritional evaluation (body mass index and albumin) were performed. Univariate analysis was performed using simple linear correlations. Regression analysis was performed using an exit level of p
Conclusions: The frequency of HS in patients with CF is high and it is not related to malnutrition, according to the parameters of BMI, TSF and MAMC. The values of MAC and MAMC indicated a greater reserve of muscle mass in patients with HS.RESUMO Objetivo: Determinar a preval ência de esteatose hepática (EH) em crianças e adolescentes com fibrose cística (FC) e associá-la com o estado nutricional. Métodos: Estudo transversal com crianças e adolescentes com diagnóstico de FC. Foram aferidos o peso e a altura para o cálculo do índice de massa corpóre...
Network study group Abstract RATIONALE: Lumacaftor-ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination recently approved for patients with cystic fibrosis (CF) homozygous for the Phe508del mutation. OBJECTIVES: To evaluate the safety and effectiveness of lumacaftor-ivacaftor in adolescents (≥12 years) and adults (≥18 years) in a real-life post-approval setting. METHODS: The study was conducted in the 47 CF reference centers in France. All patients who initiated lumacaftor-ivacaftor from January 1st to December 31st 2016 were eligible. Patients were evalua...
To evaluate whether cystic fibrosis transmembrane conductance regulator (CFTR) variants are more common among individuals tested for primary ciliary dyskinesia (PCD) compared with controls.
Reactions were much more common in patients with cystic fibrosis and those with previously identified environmental or drug allergies.Medscape Medical News