Added 9 days ago Drug news

Phase III ARRIVAL data on Kalydeco in cystic fibrosis published in The Lancet Respiratory Medicine.- Vertex Pharma.

Vertex Pharmaceuticals announced data from the ongoing Phase III, open-label ARRIVAL study and published online in The Lancet Respiratory Medicine...

Added 28 days ago Drug news

FDA approves Lokelma to treat hyperkalaemia.-AstraZeneca.

The FDA has approved Lokelma (sodium zirconium cyclosilicate), formerly ZS-9, for the treatment of adults with hyperkalaemia, a serious condition...

Added 1 month ago Drug news

Phase III study of Epidiolex in Lennox-Gastaut Syndrome published in NEJM.- GW Pharma.

The second of two phase III studies showing that Epidiolex (cannabinoid) from GW Pharma, cut seizures in patients with the...

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 6 days 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 6 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 6 days ago

Effectiveness and safety of long-term treatment with sulfonylureas in patients with neonatal diabetes due to KCNJ11 mutations: an international cohort study.

Background: KCNJ11 mutations cause permanent neonatal diabetes through pancreatic ATP-sensitive potassium channel activation. 90% of patients successfully transfer from insulin to oral sulfonylureas with...

Added 6 days ago

Chromosome 6q24-related diabetes mellitus.

Chromosome 6q24-related diabetes mellitus is the most common cause of transient neonatal diabetes (TNDM), accounting for approximately two-thirds of all TNDM cases.

Added 7 days ago

Successful off-label sulfonylurea treatment of neonatal diabetes mellitus due to chromosome 6 abnormalities.

Chromosome 6 abnormalities such as paternal uniparental isodisomy, paternal 6q24 duplication, and maternal DMR (differentially methylated region) hypomethylation are a common cause of transient neonatal diabetes mellitus (TNDM).

Added 7 days ago

Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants (HAVEN 5)

This randomized, multicenter, open-label, Phase 3 study is designed to investigate the efficacy, safety, and pharmacokinetics of emicizumab in participants with hemophilia A regardless of factor VIII (FVIII) inhibitor status.

Added 3 months ago

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A (HAVEN 4)

This multicenter, open-label, non-randomized study will assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at a dose of 6 milligrams per kilogram (mg/kg) every 4 weeks...

Added 3 months ago

Bioavailability and Pharmacokinetics of Intranasal Dexmedetomidine in Children

We aim to characterize the bioavailability and pharmacokinetics of dexmedetomidine after intranasal dosing employing pharmacometric methods in otherwise healthy 1 month to 11 years of age children scheduled...

Added 1 year ago