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Rare Diseases

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. 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. more

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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. 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. 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. 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 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. more

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