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Novartis’ blood cancer therapy Kymriah recommended by NICE for routine NHS use

In England, ALL is responsible for around 300 new diagnoses every year in people aged 25 years and under

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AbbVie’s oral therapy Aquipta recommended by NICE for migraine prevention in adults

Approximately 170,000 patients in England are expected to benefit from the agency’s decision

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Gilead’s Veklury receives NICE recommendation to treat COVID-19 in vulnerable adults and children

Veklury is now the first antiviral recommended by the agency to treat eligible children

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NICE backs post-stroke genetic testing to identify most suitable treatment options

An estimated 32% of people in the UK have at least one of the highlighted CYP2C19 gene variants

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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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Pfizer’s Velsipity receives MHRA and NICE authorisations to treat ulcerative colitis

The immune-mediated inflammatory bowel disease affects approximately 300,000 people in the UK

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Ipsen’s Cabometyx and BMS’ Opdivo recommended by NICE for advanced kidney cancer

The decision could benefit approximately 2,287 patients with renal cell carcinoma annually

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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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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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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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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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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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