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Wednesday 29 April 2026

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News

Vincenzo Nigro, coordinator of the Telethon Undiagnosed Diseases Programme

Italy leads the way on child genetic disease diagnosis

A scheme to hasten the diagnosis of children with genetic disorders is achieving strong results and could be a template for national health systems.

News

FDA clears first gene therapy for rare genetic disease WAS

Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.

Editor's Picks

FDA clears first genetic hearing loss gene therapy

Trump signs order to hasten psychedelic medicine access

UK Knowledge Quarter welcomes Boehringer Ingelheim AI accelerator

UK Biobank patient data placed on sale in China

Medicare obesity drug pilot extended after insurer pushback

News

Chiesi signs $1.9bn deal to buy KalVista and its HAE drug

Chiesi has boosted its rare disease business with an agreement to buy KalVista and Ekterly, its oral drug for treating attacks in rare disease HAE.

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Sales & Marketing

Using AI-driven synthetic personas to take your insights fur...

As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.

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