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rare diseases

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Amgen’s Uplizna shows promise in rare fibroinflammatory disease IgG4-RD

The company is planning to file for US approval of the CD19-targeted drug in this patient population

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Amgen’s interchangeable Soliris biosimilar Bkemv approved by FDA for rare blood disorders

Bkemv has been authorised to treat paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome

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Biogen to expand immunology and rare disease portfolio with $1.8bn HI-Bio acquisition

The company will gain access to HI-Bio’s lead asset for immune-mediated and rare diseases

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AstraZeneca unveils plans to deliver $80bn in total revenue by 2030

The company expects to launch 20 new medicines by the end of the decade

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Amgen’s Imdelltra granted FDA accelerated approval to treat small cell lung cancer

More than 234,000 new cases of lung cancer are expected to be diagnosed in the US this year

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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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Sandoz receives FDA approval for first denosumab biosimilars Wyost and Jubbonti

The biosimilars have been authorised for all indications covered by Amgen's reference medicines

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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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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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