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metreleptin

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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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AstraZeneca’s Voydeya receives CHMP recommendation for rare blood disease PNH

Up to 20% of PNH patients treated with a C5 inhibitor experience clinically significant extravascular haemolysis

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AstraZeneca’s Tagrisso shows promise as lung cancer maintenance therapy

Up to 85% of lung cancer patients are diagnosed with non-small cell lung cancer

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Alexion launches ‘colourUp4RARE challenge’ to mark Rare Disease Day 2024

The campaign is aimed at raising awareness of how to improve quality of life for patients

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Enhancing the patient–HCP relationship in rare diseases

By Maggie Lai

AMICULUM

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AstraZeneca’s Tagrisso combination approved by FDA for advanced lung cancer

An estimated 200,000 people in the US are diagnosed with lung cancer every year

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Navigating challenges of MedTech research with rare-disease patients

Authored by: Tom Donnelly, MedTech Director and Amanda Pirraglia, Associate Director. Published in Quirk's Magazine.

Research Partnership

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Rare disease market access – Launching orphan drugs in secondary markets: the Nordic and MENA regions

Join us live at 1.00pm (GMT)/2.00pm (CET)/8.00am (EST) on Thursday 29th February, to explore rare disease market access challenges and learn more about these high potential markets.

Mtech Access

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AstraZeneca shares positive phase 3 results for acoramidis in ATTR cardiomyopathy

Up to 500,000 people worldwide are affected by the rare and progressive condition

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Clinigen’s NaviGATE programme reveals knowledge gaps in rare disease community

There are over 7,000 rare diseases that impact more than 300 million people globally

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Sanofi expands rare disease pipeline with $2.2bn Inhibrx acquisition

The deal includes a mid-stage candidate for alpha-1 antitrypsin deficiency

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AstraZeneca’s Imfinzi combination shows promise in phase 3 liver cancer study

Hepatocellular carcinoma accounts for about 75% of all primary liver cancers in adults

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