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migalastat

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NICE recommends Genmab’s Tivdak for recurrent or metastatic cervical cancer

It is estimated that 3,300 women are diagnosed with cervical cancer in the UK each year

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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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Durbin PLC becomes Uniphar, bolstering medicine access amidst growing healthcare demands

Against the backdrop of widespread medicine shortages, Durbin PLC’s rebrand to Uniphar and integration into Uniphar | Global Sourcing represents a strategic move to address industry-wide challenges, expanding its sourcing...

Uniphar

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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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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

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Sanofi and Regeneron’s Dupixent receives positive NICE final draft guidance for COPD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

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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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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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