Blog articles

A computer manipulated photo of a hand holding a pair of self-locking medical tweezer scissors and DNA helixes. The tweezer scissors are removing a small red section that is on one of a DNA helixes.

Hereditary angioedema gene therapy hope

A CRISPR-based genome therapy has shown promising results for patients with a rare genetic swelling disorder
A row of test tubes full of neon pink liquid, each with a pipette dangling above it, drips of more pink liquid about to drop into them.

Whole genome sequencing – personalising cancer care

We explore how data from the 100,000 Genomes Project is still being used to great effect in cancer prevention and treatment

How the NHS will embed genomics into pharmacy practice

We take a closer look at the new three-year strategic framework for integrating genomic medicine into pharmacy education and training

World-first gene therapy trial for Hunter syndrome opens

We explore a new treatment that aims to cross the blood-brain barrier for the first time in Hunter syndrome
A strand of stylised blue DNA on a plain background with a magnifying glass focusing on a section of the DNA strand

Casgevy – how it works

In this week’s article, we revisit Casgevy and explore its utility and application in healthcare
An illustration of a multicoloured DNA strand running vertically down the centre of the image with two hands on either side of it. The hands are wearing blue surgical gloves. One hand is holding tweezers that are gripping the DNA strand and the other hand is holding a scalpel which has sliced the DNA strand.

Casgevy – the first CRISPR therapy

The UK has approved a new type of gene therapy for the blood disorders sickle cell disease and beta-thalassaemia, a world-first using CRISPR technology

Polygenic risk scores and DTC testing: a problematic pairing?

We explore polygenic risk scores and why their application in at-home genomic testing has been brought into question
A graphic of an ear in isolation on a blue background. There are white and grey DNA strands going diagonally across the background

Gene therapy – a new approach for paediatric hearing loss

A new gene therapy is being trialled for children with a rare form of deafness caused by variants in the OTOF gene

News articles

Rare Disease Day 2024

Educating the NHS workforce about the role of genomics in rare disease is a priority for the NHS England Genomics Education Programme

Genomic testing course runs again for Rare Disease Day 2024

Engage with expert mentors and fellow learners in the latest run of the Genomics Education Programme’s online course

Events

The entrance to the Royal Society of Medicine

The Unusual Suspects: Rare disease in everyday medicine

Medics4RareDiseases (M4RD) is proud and excited to announce details for its 11th annual symposium; The Unusual Suspects 2024: Rare Diseases in Everyday Medicine
People looking at items in glass cabinets

First, do no harm: Expert reflections on a rare bone disease

Medics4RareDiseases and FOP Friends, in collaboration with the Hunterian Museum at the Royal College of Surgeons of England, are hosting an evening reception on 13 February, for healthcare professionals