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Pfizer and Innovent Biologics agree on deal worth up to $10.5bn

The companies plan to research and develop 12 new cancer treatments

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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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Pfizer’s Braftovi combination therapy shows positive results for colorectal cancer

Colorectal cancer is the third most common cancer globally, with approximately 1.8 million people diagnosed in 2022

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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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Pfizer’s Hympavzi significantly reduces bleeds in haemophilia patients

Over 800,000 people worldwide live with haemophilia

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Pfizer’s PADCEV gets FDA approval for bladder cancer

Bladder cancer affects more than 614,000 people globally each year

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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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