Latest news articles

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 13 days ago Drug news

RESCUE Phase III trial of GS 010 failed to meet primary endpoint in Leber Hereditary Optic Neuropathy.- GenSight Biologics

GenSight Biologics announced results from the first scheduled readout, at Week 48, of the RESCUE Phase III clinical trial evaluating...

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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.

Cystic Fibrosis Knowledge Centre

Cystic Fibrosis Knowledge Centre

View disease awareness information, treatment options and European Cystic Fibrosis Society best practice guidelines.

CDK 4/6 inhibitors in metastatic breast cancer

CDK 4/6 inhibitors in metastatic breast cancer

The CDK 4/6 Inhibitors in Metastatic Breast Cancer Learning Zone provides insights and information for metastatic breast cancer (mBC) and CDK 4/6 signalling. This includes the burden and pathophysiology of the disease, the role of CDK 4/6 in cell proliferation and an overview of the CDK 4/6 inhibitors that have been approved for the management of mBC.

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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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 8 months 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 8 months ago

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Journal articles

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

Skeletal Consequences of Nephropathic Cystinosis.

Nephropathic cystinosis is a rare lysosomal storage disorder. Patients present in the first year of life with renal Fanconi syndrome that evolves to progressive chronic kidney disease (CKD).

Added 5 days ago

Ocular changes in nephropathic cystinosis: The course of the gold-dust.

In the cornea, crystalline, gold-dust deposition of cystine leads to visual impairment, recurrent erosions, photophobia, epiphora and blepharospasmus.

Added 5 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 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

Setmelanotide for the treatment of early-onset POMC deficiency obesity

The purpose of the study is to determine the effect of setmelanotide (RM-493) on weight and other factors in patients with pro-opiomelanocortin (POMC) deficiency obesity due to rare bi-allelic loss-of function POMC or PCSK1 genetic mutations.

Added 2 years 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 4 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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