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Huntinton’s disease

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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Roche opens new Swiss Institute of Human Biology research facility

The new building is part of a 1.5bn euro site investment

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UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

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Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

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Roche’s Columvi improves survival in large B-cell lymphoma

Approximately 160,000 people are diagnosed with this aggressive form of cancer each year

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Roche receives FDA clearance for whooping cough test

There are around 24.1 million cases of whooping cough annually, with around 170,000 deaths

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AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

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AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

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Genentech announces positive clinical results for BTK inhibitor MS treatment

Multiple sclerosis affects more than 2.9 million people globally

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Roche’s small cell lung cancer therapy approved by FDA

This is the first and only approved combination therapy for extensive-stage small cell lung cancer

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