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Kanuma

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Sanofi’s Xenpozyme not recommended by NICE for ultra-rare disease ASMD

An estimated 40 to 50 people in England are living with the lysosomal storage disorder

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GSK’s Jemperli combination recommended by NICE as first-line endometrial cancer treatment

Approximately 580 patients will now be eligible for treatment with the PD-1 inhibitor

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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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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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Alexion launches ‘colourUp4RARE challenge’ to mark Rare Disease Day 2024

The campaign is aimed at raising awareness of how to improve quality of life for patients

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Pfizer’s Litfulo recommended by NICE as first treatment for severe alopecia areata

Up to 14,000 patients living with the autoimmune disease could benefit from NICE’s decision

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AstraZeneca’s Tagrisso combination approved by FDA for advanced lung cancer

An estimated 200,000 people in the US are diagnosed with lung cancer every year

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GSK’s Omjjara recommended by NICE to treat myelofibrosis patients with anaemia

Myelofibrosis is estimated to affect over 1,900 people in England, Wales and Northern Ireland

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NICE recommends STADA and Calliditas’ Kinpeygo to treat rare kidney disease IgAN

Affecting approximately 14,000 in England, IgA nephropathy occurs when IgA accumulates in the kidneys, causing inflammation and scarring

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AstraZeneca shares positive phase 3 results for acoramidis in ATTR cardiomyopathy

Up to 500,000 people worldwide are affected by the rare and progressive condition

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Sobi receives NICE recommendation for Zynlonta in two non-Hodgkin lymphomas

Over 10,000 people are diagnosed with a type of non-Hodgkin lymphoma every year in the UK

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