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Rare Diseases

Over the past 10 years, the diagnoses of rare diseases in patients has cost the NHS over £3.4 billion, new research shows. Read more

News

A new funding scheme to help researchers better understand rare diseases has been announced by Great Ormond Street Hospital Children’s Charity (GOSH) and medical research charity LifeArc. Read more

News

The UK has become the first nation in the world to complete the largest genome sequencing project in healthcare, the government has announced. Read more

Opinion

The US Food and Drug Administration (FDA) has granted accelerated approval for a new treatment for adults with Fabry disease — a rare genetic disorder, resulting from the build-up of globotriaoscylceramide (GL-3) in blood vessels and organs. Read more

News

Specialist British pharmaceutical wholesaler, Chapper healthcare, has published a white paper exploring the global challenges of access to and delivery of speciality medicines. Read more

News

A new study from the University of Eastern Finland, published in the European Journal of Clinical Pharmacology, has highlighted that there are differences in the availability of orphan medicines between different European countries. Read more

Analysis

A strategic agreement has been signed by GlaxoSmithKline (GSK) and Orchard Therapeutics, involving the transfer of GSK’s portfolio of approved and investigational rare disease gene therapies to Orchard. Read more

News

This year will be big for Duchenne muscular dystrophy (DMD) as interest in treatments is on the rise and deal activity is increasing, reported data and analytics company, GlobalData. Read more

Analysis

Sanofi and RNAi therapeutics company, Alnylam Pharmaceuticals, have begun strategic restructuring of their RNAi therapeutics alliance to streamline and optimise development and commercialisation of certain products Read more

News

A consultation by NICE and NHS England on changes to the arrangements for evaluating and funding medicines could potentially stop the flow of new medicines reaching patients with rare and complex diseases. Read more

News