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Clinuvel Pharmaceuticals

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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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AstraZeneca reports positive phase 3 data for tozorakimab in COPD

COPD affects nearly 400 million people globally and is the third leading cause of death worldwide

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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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Astellas’ application to EMA validated for treatment of muscle-invasive bladder cancer

Bladder cancer affects over 224,000 people annually in Europe, with over 30% of cases being muscle-invasive bladder cancer

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EMA’s PRIME scheme launches three major new features

The scheme supports the development of treatments targeting unmet medical needs in the EU

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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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AstraZeneca’s Imfinzi approved in EU for early gastric and gastro-oesophageal cancers

This is the first and only perioperative immunotherapy approved to treat early gastric cancers

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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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AstraZeneca’s Imfinzi regimen receives positive CHMP opinion for early gastric and gastroesophageal cancer

Gastric cancer is the fifth highest cause of cancer deaths worldwide, with almost a million people diagnosed annually

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Sanofi’s Rezurock receives positive CHMP opinion for chronic graft-versus-host disease

Up to 50% of allogeneic haematopoietic stem cell transplant patients develop chronic GVHD

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AstraZeneca and CSPC Pharmaceuticals enter collaboration on weight management therapies

Almost three billion people globally are thought to be either overweight or obese

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