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metreleptin

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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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InflaRx seeks FDA authorisation for vilobelimab for critically ill COVID-19 patients

A phase 3 study showed that the antibody reduced the all-cause mortality rate after 28 days

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Amylyx’s Relyvrio receives FDA approval for ALS treatment

Relyvrio was shown to significantly slow physical decline in people living with ALS

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Sanofi and Regeneron’s Dupixent approved by FDA for prurigo nodularis

The two phase 3 trials showed a meaningful reduction in itching

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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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