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Rare Disease Day 2023 – raising awareness around the world

Around one in 17 people will be affected by a rare disease at some point in their lives

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Sanofi reports positive new data for multiple sclerosis treatment tolebrutinib

It is estimated that there are over 130,000 people living with the condition in the UK alone

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Sanofi and Sobi’s once-weekly haemophilia A treatment approved by FDA

Current factor VIII therapies usually require patients to receive treatment every two days

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European Commission grants AstraZeneca three new approvals

The indications are for adults with liver and lung cancers, and chronic lymphocytic leukaemia

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AstraZeneca/Sanofi’s Beyfortus shows consistent protection against RSV disease in infants

RSV is the most common cause of infant respiratory infections, including pneumonia

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EC approves first gene therapy for adults with haemophilia B

Typically, haemophilia B patients must adhere to strict, lifelong IV infusion schedules

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EC approves UCB’s Fintepla to treat severe form of epilepsy

Lennox-Gastaut syndrome affects around two in 10,000 people in the EU and usually begins in early childhood

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AstraZeneca’s heart failure treatment approved for expanded use in EU

Chronic heart failure is the leading cause of hospitalisation for those over the age of 65

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Sanofi’s enzyme replacement therapy available on NHS to Pompe disease patients

The rare genetic disorder affects around one in every 40,000 people in the UK

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Sanofi/Regeneron’s Dupixent approved by EC for eosinophilic oesophagitis

This makes Dupixent the first and only targeted medicine indicated for this condition

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Sanofi/Regeneron’s Dupixent recommended by CHMP for atopic dermatitis in children

If approved, the medicine would be the first and only targeted option in the EU for this age group

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Sandoz’s high concentration Hyrimoz formulation recommended for use in EU

The recommendation includes multiple indications such as rheumatoid arthritis and Crohn’s disease

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