Data from multiple sources - Curated by Marshall Pearce - Last updated 06 June 2017

Latest news articles

Added 6 days ago Drug news

Resumption of dosing in clinical studies of fitusiran for hemophilia A & B

Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced a successful Type A meeting with the FDA.

Added 14 days ago Drug news

Phase III trial of Samsca slows kidney function decline in polycystic kidney disease.- Otsuka.

In the phase III, multi-center, randomized withdrawal, placebo controlled, double-blind REPRISE trial Samsca (tolvaptan), from Otsuka, slowed kidney function decline...

Added 15 days ago Drug news

Results from a study on Procysbi delayed-release capsules in treatment-naïve children younger than 6 years of age with nephropathic cystinosis.- Horizon Pharma.

Horizon Pharma plc announced results from a new open-label study evaluating the effects of Procysbi (cysteamine bitartrate) delayed-release capsules in...

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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

Cystic Fibrosis

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

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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.

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Guidelines

Canadian Guidelines for the Management of Plaque Psoriasis 2009

Since the publication of the 2009 Canadian Guidelines for the Management of Plaque Psoriasis, significant advances have been made in determining the role of T cells in inflammatory and autoimmune diseases. 

Added 9 months ago

European S3-Guidelines on the systemic treatment of psoriasis vulgaris

In recognition of the impact of psoriasis on patients the World Health Assembly (WHA) in May 2014 supported including psoriasis into the group of non-communicable diseases.

Added 9 months ago

Familial hypercholesterolaemia in children and adolescents: gaining decades of life by optimizing detection and treatment

Familial hypercholesterolaemia (FH) is a common genetic cause of premature coronary heart disease (CHD). Globally, one baby is born with...

Added 2 years ago

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Journal articles

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

Background: Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies...

Added 5 days ago

Sapropterin treatment does not enhance the health-related quality of life of patients with phenylketonuria and their parents.

Aim: Sapropterin causes reductions in blood phenylalanine concentrations in sensitive patients with phenylketonuria (PKU). We examined whether the subsequent relaxation of dietary restrictions influenced the quality of life (QoL)...

Added 9 days ago

Sapropterin dihydrochloride for phenylketonuria.

Objectives: To assess the safety and efficacy of sapropterin dihydrochloride in lowering blood phenylalanine concentration in people with phenylketonuria.

Added 9 days ago

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Clinical trials

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

A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 96 weeks, in patients with CLN2.

Added 4 months ago

Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome

To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.

Added 3 years 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 3 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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