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rare diseases

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AstraZeneca shares positive late-stage results for Imfinzi and Lynparza in endometrial cancer

The incidence of endometrial cancer is expected to increase by approximately 61% in 2050

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AstraZeneca expands rare disease pipeline with $1.05bn Amolyt Pharma acquisition

The deal gives the company access to a late-stage candidate being evaluated for hypoparathyroidism

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AstraZeneca announces plans to invest £650m in UK to boost life sciences sector

The investment will also help to enhance public health protection and pandemic preparedness in the UK

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AstraZeneca/Daiichi Sankyo’s Enhertu not recommended by NICE for advanced breast cancer

Breast cancer is the most common form of cancer in the UK, accounting for over 55,000 cases every year

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Novartis shares positive results for spinal muscular atrophy gene therapy Zolgensma

The company said the results support the use of the one-time therapy in older and heavier children

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Radboud researchers use new method to improve diagnosis of rare diseases

Approximately 7,000 rare diseases collectively impact up to 5.9% of the global population

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OneChain-led consortium granted €1.9m to advance CAR-T therapy for rare leukaemia

Acute lymphoblastic leukaemia has an incidence of around 1.5 cases per 100,000 people

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Clinigen launches ‘What is Possible?’ campaign on Rare Disease Day to accelerate access to medicines

There are over 7,000 rare diseases impacting more than 300 million people globally

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ICR study reveals AstraZeneca’s ceralasertib sensitises tumours to immunotherapy

Results showed that the drug stopped tumour growth in more than half of patients

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Novo Nordisk and Neomorph enter molecular glue degrader partnership worth over $1.4bn

The companies will work on therapies for cardiometabolic and rare diseases

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AstraZeneca’s Voydeya receives CHMP recommendation for rare blood disease PNH

Up to 20% of PNH patients treated with a C5 inhibitor experience clinically significant extravascular haemolysis

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AstraZeneca’s Tagrisso shows promise as lung cancer maintenance therapy

Up to 85% of lung cancer patients are diagnosed with non-small cell lung cancer

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