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siltuximab

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Johnson & Johnson receives updated NICE recommendation for Tecvayli in multiple myeloma

Approximately 5,900 new cases of the blood cancer are diagnosed every year in the UK

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J&J’s Erleada shows overall survival benefit in head-to-head prostate cancer study

Approximately 300,000 new cases of the disease are diagnosed in the US every year

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NHS England begins newborn screening programme to identify rare genetic conditions

The Generation Study is aiming to screen 100,000 newborns for more than 200 diseases

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J&J and Legend share long-term results for Carvykti in phase 3 multiple myeloma study

More than 35,000 people are expected to be diagnosed with the blood cancer in the US this year

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FDA approves IntraBio’s Aqneursa to treat Niemann-Pick disease type C patients

The rare lysosomal storage disorder affects approximately one in every 100,000 to 120,000 births

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Johnson & Johnson’s Yuvanci granted EC approval to treat pulmonary arterial hypertension

Yuvanci is now the only single tablet combination therapy for PAH patients in Europe

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MHRA approves Pharming’s Joenja as first drug for rare immune disease APDS

Up to 40 people in the UK are affected by activated phosphoinositide 3-kinase delta syndrome

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FDA grants rare paediatric disease designation to Enterprise’s cystic fibrosis candidate

The genetic disease is estimated to affect more than 100,000 people globally

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Championing people-powered communications in rare diseases: AMICULUM’s feature in The Guardian Rare Diseases Campaign 2024

By Ellie Thomas

Amiculum

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J&J’s Rybrevant combination approved by FDA to treat EGFR-mutated NSCLC

Alterations in EGFR are among the most common driver mutations in lung cancer patients

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Novo Nordisk and NanoVation to develop genetic medicines in partnership worth $600m

The companies will collaborate on up to seven programmes for cardiometabolic and rare diseases

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J&J’s Rybrevant combination shows positive overall survival trend in late-stage lung cancer study

Alterations in EGFR are among the most common driver mutations in NSCLC patients

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