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AstraZeneca’s Tagrisso combination receives CHMP recommendation for advanced lung cancer

More than 450,000 people are diagnosed with lung cancer every year in Europe

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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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CHMP recommends Valneva’s chikungunya vaccine for use in adults

If approved, Ixchiq will become the first vaccine available in the EU for the mosquito-borne viral disease

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Pfizer’s one-time haemophilia B therapy Durveqtix receives CHMP recommendation

More than 38,000 people worldwide are currently affected by the inherited bleeding disorder

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Takeda’s enzyme replacement therapy recommended by CHMP for rare blood clotting disorder cTTP

If approved, rADAMTS13 will be the first and only enzyme replacement therapy in the EU indicated for cTTP

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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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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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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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