Added 2 days ago Drug news

FDA approves Symdeko for use in children with cystic fibrosis who have two copies of the F508del-CFTR mutation or who at least one mutation in the CFTR gene.- Vertex

Vertex Pharmaceuticals Incorporated announced the FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6...

Added 3 days ago Drug news

FDA accepts sBLA for Ultomiris to treat atypical hemolytic uremic syndrome (aHUS). -Alexion

Alexion Pharmaceuticals, Inc. announced that the FDA has accepted for priority review the company’s supplemental Biologics License Application (sBLA) for...

Added 10 days ago Drug news

Phase IIIb DIALIZE trial resultsof Lokelma for hyperkalaemia in patients with end-stage renal disease on haemodialysis

AstraZeneca presented positive results from the Phase IIIb DIALIZE trial which investigated the efficacy and safety of Lokelma (sodium zirconium...

Lyme disease

This guideline covers diagnosing and managing Lyme disease. It aims to raise awareness of when Lyme disease should be suspected and ensure that people have prompt and consistent diagnosis and treatment.

Added 1 year ago

Epilepsies: diagnosis and management

The guideline covers diagnosing, treating and managing epilepsy and seizures in children, young people and adults in primary and secondary care.

Added 7 years ago

Otitis media (acute): antimicrobial prescribing

This guideline sets out an antimicrobial prescribing strategy for acute otitis media (ear infection). It aims to limit antibiotic use and reduce antimicrobial resistance.

Added 1 year ago

Inhaled hypertonic saline in preschool children with cystic fibrosis (SHIP): a multicentre, randomised, double-blind, placebo-controlled trial.

Background: Inhaled hypertonic saline enhances mucociliary clearance, improves lung function, and reduces pulmonary exacerbations in people with cystic fibrosis older than age 6 years. We aimed to assess the effect of inhaled...

Added 5 days ago

Ten years of chest MRI for patients with cystic fibrosis : Translation from the bench to clinical routine.

Background: Despite recent advances in our knowledge about the pathophysiology and treatment of cystic fibrosis (CF), pulmonary involvement remains the most important determinant of morbidity and mortality in patients with CF.

Added 6 days ago

Vitamin E status and its determinants in patients with cystic fibrosis.

Purpose: The risk of vitamin E deficiency is of primary concern in cystic fibrosis patients. However, early diagnosis and routine vitamin E supplementation can lead to its normal or even high levels. In the present study, we...

Added 6 days ago

A Study to Evaluate the Safety, Tolerability, and Efficacy of BIIB017 (Peginterferon Beta-1a) in Pediatric Participants for the Treatment of Relapsing-Remitting Multiple Sclerosis

This study will evaluate the safety, tolerability, and descriptive efficacy of BIIB017 in pediatric participants with relapsing-remitting multiple sclerosis (RRMS) and to assess the pharmacokinetics (PK) of BIIB017 in pediatric participants...

Added 15 days ago

JAK1 Inhibitor With Medicated Topical Therapy in Adolescents With Atopic Dermatitis (JADE TEEN)

This is a randomized, double blind, placebo controlled, parallel group, Phase 3 study to evaluate the efficacy and safety of PF 04965842 in adolescent participants 12 to <18 years of age with moderate to severe AD

Added 3 months ago

Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Disease

Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's disease.

Added 8 days ago