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Genomics England

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LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

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Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

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Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

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AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

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AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

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NHS programme to give head and neck cancer patients fast-track access to mRNA vaccine

More than 11,000 people are diagnosed with head and neck cancers every year in England

Navigating NHS Market Access: Policy Change and Opportunity in England With Sarah Everest Ford

Navigating NHS Market Access: Policy Change and Opportunity in England

With the changes at NHS England and the publishing of the 10-Year Plan, there is a lot of talk of change in the NHS in England. But what do these...

Petauri Evidence

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Digital Transformation in the NHS’s 10-Year Plan: Techno-Optimism and the Limits of Efficiency

Here, Evelyne Priestman (Senior Consultant – HEOR) and Mary Baker (Senior Medical Writer) at Petauri Evidence explore the National Health Service (NHS) in England’s newly unveiled 10-year plan through the...

Petauri Evidence

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