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rare diseases

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EMA grants AZ’s Vaxzevria COVID-19 vaccine full marketing authorisation

Vaxzevria has been shown to be effective against all forms of COVID-19

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Alexion’s Ultomiris shows promise in neuromyelitis optica spectrum disorder phase 3 trial

Ultomiris-treated patients showed zero relapses with a median treatment duration of 73 weeks

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AstraZeneca’s two advanced breast cancer trials meet primary endpoints

Both trials showed significant improvement in progression-free survival

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AstraZeneca’s Imjudo/Imfinzi combination approved by FDA for unresectable liver cancer

Phase 3 trial results demonstrated a 22% reduction in risk of death versus sorafenib

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FDA awards over $38m for advancement of rare disease treatments

Grants will support clinical trials, natural history studies and regulatory science tools

Patient Centric Approaches for Rare and Ultra-Rare Diseases

Lawrence Bressler, Head of Value & Market Access, Global Rare Diseases at Chiesi, shares how Chiesi is revolutionizing the lives of people living with rare diseases on a global scale....

Impetus Digital

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Reframing rare to increase earlier diagnosis of Gaucher Disease

Sanofi Genzyme wanted to reach haematologists; the specialist physicians most likely to encounter patients with Gaucher Disease prior to diagnosis. The problem? Gaucher Disease is considered very rare and its...

Create Health

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Rare diseases: making a difference through education

Medscape Education team up with The Guardian to launch a Rare Disease campaign in the UK.

Medscape Education Global

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AstraZeneca’s Tezspire receives MHRA approval as add-on treatment for severe asthma

Tezspire demonstrated reduced annualised rate of asthma exacerbations

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AstraZeneca’s Tezspire receives EC approval as severe asthma add-on treatment

Tezspire demonstrated superiority across every primary and key secondary endpoint compared to placebo

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AstraZeneca’s COVID-19 treatment granted EC approval

Evusheld significantly reduced risk of severe COVID-19 or death in a phase 3 trial

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BeiGene’s Brukinsa gets green light from NICE as Waldenström’s macroglobulinemia treatment

The condition occurs in less than 2% of patients with non-Hodgkin lymphomas

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