Added 3 days ago Drug news

Phase III study for tezacaftor/ivacaftor combination in cystic fibrosis supports European Medicines Agency submission.- Vertex

Vertex Pharmaceuticals Incorporated announced the results of a Phase III study conducted in Europe and Australia of tezacaftor in combination...

Added 11 days ago Drug news

Fintepla filed with FDA and MAA for Dravet syndrome.- Zogenix

Zogenix has completed its rolling submission of a New Drug Application (NDA) to the FDA and submitted a Marketing Authorization...

Added 20 days ago Drug news

Phase III success for Ultomiris in complement inhibitor-naïve patients with atypical hemolytic uremic syndrome . Alexion Pharma

Alexion Pharmaceuticals, Inc. announced that the Phase III study of Ultomiris (ravulizumab-cwvz), the company’s long-acting C5 complement inhibitor, met its...

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 8 months 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 8 months ago

Teenagers and young adults with nephropathic cystinosis display significant bone disease and cortical impairment.

Background: Bone impairment appears to be a novel complication of nephropathic cystinosis despite cysteamine therapy. Its exact underlying pathophysiology is nevertheless unclear.

Added 5 days ago

Nephropathic cystinosis: an update.

In this review, we aim to provide an overview of the latest advances in the pathogenetic, clinical, and therapeutic aspects of cystinosis.

Added 5 days ago

Nephropathic Cystinosis: Symptoms, Treatment, and Perspectives of a Systemic Disease.

Cystinosis is a rare autosomal recessive lysosomal storage disorder caused by mutations in the CTNS gene. Main dysfunction is a defective clearance of cystine from lysosomes that leads to accumulation of cystine crystals in every tissue of the body.

Added 5 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 11 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 11 months ago

Study of S-033188 (Baloxavir Marboxil) Compared With Placebo or Oseltamivir in Patients With Influenza at High Risk of Influenza Complications (CAPSTONE 2)

The primary objective of this study is to evaluate the efficacy of a single, oral dose of Baloxavir Marboxil compared with placebo by measuring the time to improvement of influenza symptoms in patients with influenza...

Added 7 months ago