Latest news articles

Added 10 days ago Drug news

New phase III results with Fintepla (ZX 008) in Dravet Syndrome presented.- Zogenix.

Zogenix announced that data will be presented on the use of Fintepla (low-dose fenfluramine), in children and young adults with...

Added 10 days ago Drug news

New data from Phase 1/II HGB-206 study of LentiGlobin gene therapy in patients with sickle cell disease.-bluebird bio.

bluebird bio, Inc. announced new data from patients in Group C of its ongoing Phase 1/II HGB-206 study of the...

Added 10 days ago Drug news

Results of Phase III trials NORTHSTAR-2 and NORTHSTAR-3 of LentiGlobin gene therapy to treat transfusion-dependent beta-thalassemia. bluebirdbio.

bluebird bio, Inc. announced new data from the Phase III Northstar-2 (HGB-207) and Northstar-3 (HGB-212) clinical studies of its investigational...

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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 6 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 5 months ago

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Journal articles

A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease.

Drug development for ultra-rare diseases is challenging because small sample sizes and heterogeneous study populations hamper the ability of randomized, placebo-controlled trials with a single primary endpoint to demonstrate valid treatment effects.

Added 3 months ago

The Genetics of Chronic Itch: Gene Expression in the Skin of Patients with Atopic Dermatitis and Psoriasis with Severe Itch.

To identify itch-related mediators and receptors that are differentially expressed in pruritic skin, we used RNA sequencing to analyze the complete transcriptome in skin from paired itchy, lesional and nonitchy...

Added 4 months ago

Nusinersen: A Treatment for Spinal Muscular Atrophy.

Objective: To review the efficacy and safety of nusinersen (Spinraza) in the treatment of spinal muscular atrophy (SMA).

Added 4 months 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 9 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 9 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

Association of adherence to lifestyle recommendations and risk of colorectal cancer: a prospective Danish cohort study

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The diagnostic value of hyperammonemia induced by the non-ischemic forearm exercise test

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BMJ Learning - British Medical Learning
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Carcinoma of unknown primary origin: diagnosis and management: putting NICE guidelines into practice

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Union of European Medical Specialists (via European Accreditation Council for CME)
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