Latest news articles

Added 1 day ago Drug news

UK's MHRA has renewed for a further year the Early Access to Medicines Scheme scientific opinion for idebenone for patients with Duchenne muscular dystrophy.- Santhera

Santhera Pharmaceuticals announces that the UK's Medicines and Healthcare products Regulatory Agency (MHRA) has renewed for a further year the...

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 10 days ago Drug news

Phase III HOPE Study of GBT 440 met primary endpoint in sickle cell disease

Global Blood Therapeutics announced new results from its Phase III HOPE Study of GBT 440 (voxelotor) in patients ages 12...

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Learning Zones

An epgonline.org Learning Zone (LZ) is an area of the site dedicated to providing detailed self-directed medical education about a disease, condition or procedure.

Chronic Lymphocytic Leukaemia (CLL)

Chronic Lymphocytic Leukaemia (CLL)

Refine your knowledge of chronic lymphocytic leukaemia (CLL) with information on pathophysiology, diagnosis, treatment options and more

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Transplantation

Transplantation

See information on best practice in solid organ transplantation, and expert discussions on related hot topics.

Psoriasis

Psoriasis

See information on psoriasis pathophysiology, signs and symptoms, comorbidities, treatment options, and more.

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Guidelines

Cystic fibrosis: diagnosis and management

This guideline covers diagnosing and managing cystic fibrosis. It specifies how to monitor the condition and manage the symptoms to improve quality of life.

Added 1 year ago

British Association of Dermatologists guidelines for biologic therapy for psoriasis 2017

The overall aim of the guideline is to provide evidence-based recommendations on the use of biologic therapies (adalimumab, etanercept, infliximab, ixekizumab, secukinumab and ustekinumab) in adults, children and young people...

Added 9 years ago

Clinical practice guidelines for the care of girls and women with Turner syndrome: proceedings from the 2016 Cincinnati International Turner Syndrome Meeting.

Turner syndrome affects 25-50 per 100,000 females and can involve multiple organs through all stages of life, necessitating multidisciplinary approach to care.

Added 1 year ago

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Journal articles

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

Augmentation of CFTR function in human bronchial epithelial cells via SLC6A14-dependent amino acid uptake: Implications for treatment of Cystic Fibrosis.

Rationale: SLC6A14 mediated L-arginine transport has been shown to augment the residual anion channel activity of the major mutant, F508del-CFTR, in the murine gastrointestinal tract. It is not yet known if this transporter...

Added 6 days ago

Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries.

Background: Ivacaftor is the first in a class of drugs, CFTR modulators, that target the underlying defect in cystic fibrosis (CF). This long-term observational safety study evaluated CF disease progression in patients treated...

Added 6 days ago

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Clinical trials

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 1 year ago

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria (PKU)

This study will evaluate SYNB1618 (investigational product) in healthy volunteers (HV) and subjects diagnosed with phenylketonuria (PKU) for the possible treatment of PKU. Phenylketonuria (PKU) is a rare inherited metabolic...

Added 2 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 1 year ago

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Blog Posts

Hugh Harvey

Metrics Madness

Posted 3 years ago

You shouldn't judge a book by its cover. That much is true, but it's also common sense not to judge a book by measuring the whiteness of its pages, or the amount of hamsters you can place on it. So why do we measure the fabulous NHS with nonsense metrics?

Kimberley Kendall

An Introduction to Complex Disease Genetics

Posted 5 years ago

What's the difference between a gene 'for' a disease and genes that predispose you to susceptibility? How can we use markers to determine the likelihood a patient will develop a disease? What's nature and what's nurture?

Dr Kimberley Kendall introduces the complexities of Disease Genetics. Affecting the whole of medicine, disease genetics are already revolutionising how we think about medicine. We welcome your views in the comments.

CME

Congenital heart disease in adults

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Easily missed: Hereditary haemochromatosis

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Breaking bad news: a how to do it guide

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