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- PMLiVE

Incyte Biosciences UK announces NICE approval for Opzelura for vitiligo

This is the first approved treatment option in the UK for patients with non-segmental vitiligo

- PMLiVE

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

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

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

- PMLiVE

Sanofi and Regeneron’s Dupixent receives positive NICE final draft guidance for COPD

Around 1.4 million people in the UK are diagnosed with COPD, with two million thought to be undiagnosed

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

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UK and US agree on landmark pharma pricing tariff deal

US tariffs on UK-produced pharmaceuticals will remain at zero for three years

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AstraZeneca announces $2bn investment in US manufacturing

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

- PMLiVE

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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ViiV Healthcare receives NICE recommendation for HIV prevention therapy

The treatment is the first and only long-acting HIV prevention option available on the NHS in England and Wales

- PMLiVE

Pfizer’s Lorviqua recommended by NICE for lung cancer

Lung cancer is the leading cause of cancer-related deaths worldwide

- PMLiVE

New National Commission launched to strengthen AI regulation

The Commission will unite healthcare leaders, regulators and technology experts to shape future AI governance

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