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,LALD Header

The Lysosomal Acid Lipase (LAL) Deficiency Knowledge Centre is intended to provide healthcare professionals with accurate disease awareness and treatment information for patients with this condition.

LAL deficiency is an autosomal recessive lysosomal storage disorder resulting in marked lysosomal accumulation of cholesteryl esters and triglycerides. LAL deficiency has multi-organ involvement including the liver, spleen, and cardiovascular system. The two major phenotypes of this condition are a rapidly progressive form in infancy (formerly known as Wolman disease) and a later-onset progressive form in children and adults (formerly known as cholesteryl ester storage disease, CESD).

Users of this educational resource can expect to find clinical content arranged within three main sections:

Disease Awareness: including epidemiology, pathophysiology, genotypic presentation, diagnosis, prognosis and more…

Treatment: presents the drug characteristics, safety and efficacy of sebelipase alfa – the first and only licenced treatment for LAL deficiency

Resources: useful external links, events calendar and a video channel with short expert video discussions on topics such as diagnostic approach and current prognosis.

Developed by EPG Health Media (Europe) Ltd for epgonline.org, in collaboration and with content provided by Alexion Pharma GmbH

,Cystic Fibrosis Knowledge Centre

Cystic fibrosis (CF) is a genetic condition caused by a defect in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel. In people with CF, the protein does not work effectively, resulting in thick, sticky secretions at epithelial membranes – the underlying defect in CF.1 Cystic fibrosis can have multi-organ involvement with a range of complications and comorbidities reported.

The aim of the Cystic Fibrosis Knowledge Centre is to provide healthcare professionals with accurate clinical information including disease awareness information, emerging and established treatment options and current best practice based on recently updated guidelines by the European Cystic Fibrosis Society (ECFS). A resources section contains external links to upcoming medical conferences and patient support groups.

This resource is developed by EPG Health Media for epgonline.org, supported by an independent educational grant from Vertex.

1. Boucher RC. Cystic Fibrosis. In: Kasper DL, Braunwald E, Fauci AS, Hauser SL, Longo DL, Jameson JL et al., editors. Harrison's Internal Medicine. 16th ed. New York: McGraw-Hill; 2005. p. 1-9.​

Latest News

Medical Videos

An Animation of The Hepatitis C Virus and How it Affects the Body
An Animation of The Hepatitis C Virus and How it Affects the Body
Discussion of Hepatitis C in the UK House of Lords June 2011
Discussion of Hepatitis C in the UK House of Lords June 2011

Recent Drug Updates

Medical Journal Abstracts on Hepatology

Radiofrequency ablation of hepatocellular carcinoma: Mono or multipolar?

Tue 01 Mar 2016 -  Radiofrequency ablation of hepatocellular carcinoma: Mono or multipolar?

Background and aims: Thermo-ablation by radiofrequency is recognized as a curative treatment for early-stage hepatocellular carcinoma. However, local recurrence may occur because of incomplete ...

Hepatocellular Carcinoma in Obesity, Type 2 Diabetes, and NAFLD.

Fri 26 Feb 2016 -  Hepatocellular Carcinoma in Obesity, Type 2 Diabetes, and NAFLD.

Hepatocellular carcinoma (HCC) is the second commonest cause of cancer death worldwide. Rather than falling as a result of prevention and treatments for viral hepatitis, an increase is evident in ...

Clinical Guidelines

United Kingdom National Guideline on the Management of the viral hepatitides A, B and C 2015

Jan 2016

This guideline is an update of the guideline published in 2008 and, like its previous version,..

... provides guidance for best practice in the diagnosis, management and prevention of viral hepatitis types A, B and C. It is primarily intended for use in UK Genitourinary Medicine (GUM)/Sexual Health settings, but can be applied or adapted for use in other settings where sexually transmitted infection (STI) assessments are undertaken such as in integrated STI/contraception sexual health services and other specifically commissioned services, including general practice. This guideline is for use by all healthcare professionals (doctors, nurses and health advisers) and for use when dealing with patients who are attending with symptoms that may be attributed to a STI, with concern about STIs or are requesting screening for STIs. It will also be useful to managers and commissioners of sexual health services in understanding service needs.

The purpose of the guideline is to help improve the sexual health of individuals attending sexual health clinics by encouraging high standards of care. The guideline offers recommendations on best practice regarding viral hepatitis for both men and women, including adolescents.

EASL-ALEH Clinical Practice Guidelines: Non-invasive tests for evaluation of liver disease severity and prognosis

Apr 2015

These Clinical Practice Guidelines (CPGs) have been developed by a panel of experts chosen by the..

... EASL and ALEH Governing Boards. The recommendations were peer-reviewed by external expert reviewers and approved by EASL and ALEH Governing Boards.

Clinical Trials

Association Between Autologous Transfusion and Recurrence Among Patients With HCC After Resection (AT/HCC)

Fri 01 Jan 2016

This prospective study aims to determine whether autotransfusion of red blood cells salvaged during liver resection is associated with the recurrence of hepatocellular ...

Effects of Empagliflozin on Liver Fat Content, Energy Metabolism and Body Composition in Patients With Type 2 Diabetes (EmLiFa)

Tue 01 Dec 2015

The effects of empagliflozin treatment on hepatocellular lipid content, liver energy metabolism and body composition will be investigated in a multicentre, prospective, placebo-controlled, ...

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