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LEO Pharma acquires rights for dersimelagon from Tanabe Pharma

The company will pay $435m in up front and near-term milestone payments

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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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Amgen agrees to lower prescription drug costs in deal with US government

The company has invested $40bn in US R&D and manufacturing in the past seven years

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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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Amgen reports positive results from landmark cardiovascular study

The study found significant reductions of major cardiovascular events in high-risk adults following treatment

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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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