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Aide Health

- PMLiVE

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

- PMLiVE

Shionogi to acquire IntraBio in deal worth $2bn

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

- PMLiVE

Ogsiveo recommended by NICE to treat rare soft tissue tumours

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

- PMLiVE

LEO Pharma acquires rights for dersimelagon from Tanabe Pharma

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

- PMLiVE

Ultomiris gets US FDA Priority Review for immunoglobulin A nephropathy treatment

If approved, this would be the first C5 complement inhibitor available for this rare kidney disease

- PMLiVE

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

- 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

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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

- 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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