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Access The Vector Issue 3: 15 payers describe the likely affect of EU General Pharmaceutical Legislation on orphan drug strategy

In the third issue of The Vector, Genesis Research asked 15 payers across France, Germany, Italy, Spain, and the UK how they believe the new EU General Pharmaceutical Legislation will...

Genesis Research Group

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Alexion and Aide Health launch app for people with rare diseases

More than 3.5 million people in the UK live with a rare condition

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Shionogi to acquire IntraBio in deal worth $2bn

The deal will expand Shionogi’s growing portfolio with IntraBio’s rare diseases capabilities

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Ogsiveo recommended by NICE to treat rare soft tissue tumours

Desmoid tumours are rare soft tissue tumours that can grow aggressively into nearby tissues

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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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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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