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metreleptin

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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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Pfizer and BioNTech seek FDA authorisation for COVID-19 booster for children

An application will also be submitted to the European Medicines Agency to extend the marketing authorisation for this age group

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Lilly’s Retevmo approved by FDA for RET fusion+ advanced/metastatic solid tumours

Results demonstrated an overall response rate of 44% across multiple tumour types

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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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bluebird bio’s Skysona approved by FDA to treat cerebral adrenoleukodystrophy

The gene therapy was shown to slow the progression of neurologic dysfunction

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AstraZeneca/Sanofi’s Beyfortus receives CHMP recommendation for prevention of RSV disease in infants

Beyfortus reduced the incidence of medically attended lower respiratory tract infections caused by RSV

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AstraZeneca’s COVID-19 treatment receives CHMP recommendation for use in EU

Evusheld provided statistically significant protection against progression to severe COVID-19 or death

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FDA and NIHR launch public-private partnership for rare neurodegenerative diseases

The announcement follows President Biden signing the Accelerating Access to Critical Therapies for ALS Act in December 2021

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AstraZeneca’s Imfinzi plus chemotherapy shows promise for advanced biliary tract cancer

Updated results from a phase 3 trial showed the combination treatment reduced the risk of death by 24% versus chemotherapy alone

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