Latest news articles

Added 12 hours ago Drug news

FDA approves Kalydeco to treat cystic fibrosis in children aged 12 to <24 months.- Vertex Pharma.

Vertex Pharmaceuticals Incorporated announced the FDA approved Kalydeco (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12...

Added 4 days ago Drug news

FDA grants accelerated approval for Galafold treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.--Amicus Therapeutics.

Amicus Therapeutics has announced that the FDA has granted accelerated approval of Galafold (migalastat) 123 mg capsules. Galafold is an...

Added 8 days ago Drug news

FDA approves Orkambi to include use in children ages 2 through 5 years with cystic fibrosis who have two copies of the F508del-CFTR mutation.- Vertex Pharma.

Vertex Pharmaceuticals Incorporated announced the FDA has approved Orkambi (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years...

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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.

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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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 2 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 8 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 month ago

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Journal articles

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 1 day 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 27 days ago

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.

Background: Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin.

Added 1 month 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 5 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 5 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 1 year ago

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Blog Posts

Hugh Harvey

Metrics Madness

Posted 2 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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Carcinoma of unknown primary origin: diagnosis and management: putting NICE guidelines into practice

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