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activated phosphoinositide 3-kinase delta syndrome

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MHRA approves Leqselvi for severe alopecia areata

Around 30% of patients saw 80% more scalp hair after 24 weeks of treatment

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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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Novo Nordisk’s Wegovy higher dose injection approved by UK MHRA for obesity

Obesity affects approximately 15 million people in the UK

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GSK’s Nucala approved by MHRA for uncontrolled COPD

Symptoms of COPD include breathlessness, persistent cough and frequent chest infections

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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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AstraZeneca announces $2bn investment in US manufacturing

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

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Biogen and Eisai receive MHRA approval for updated dosing of early Alzheimer’s treatment

The MHRA approval for IV dosing comes following positive results from the global phase 3 trial

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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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MHRA marks 100 years of patient safety through biological standards

The UK regulator highlights its century-long role in establishing safety and consistency benchmarks

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MHRA and FDA expand collaboration on AI and medtech regulation

National Commission on the Regulation of AI in Healthcare brings together UK and US experts

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