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PureTech’s deupirfenidone gets FDA and EC Orphan Drug Designations for idiopathic pulmonary fibrosis

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or fewer than five in 10,000 individuals in the European Union

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Diabeloop’s DBLG2 receives FDA and CE clearance for automated insulin delivery without meal input

While a lack of meal announcements can lead to lower glycaemic control, it reduces the mental burden for those with type 1 diabetes

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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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Johnson & Johnson’s Darzalex Faspro regimen receives FDA approval for multiple myeloma

Multiple myeloma has an average five-year survival rate of 59.8% and is currently incurable

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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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AstraZeneca announces $15bn investment in Chinese manufacturing and R&D by 2030

The pharma company will be the first global biopharma with end-to-end cell therapy capabilities in China

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Novartis’ ianalumab receives FDA Breakthrough Therapy designation for Sjögren’s disease

Approximately half of those with Sjögren’s disease are thought to be undiagnosed

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AstraZeneca’s Saphnelo demonstrates positive results in systemic lupus erythematosus

Over 3.4 million people globally are affected by SLE, and it is one of the leading causes of death in young women in the US

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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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AstraZeneca’s Enhertu combination approved by FDA for HER2-positive breast cancer

Around one in five breast cancer cases are HER2-positive

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