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Kygevvi

- PMLiVE

LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

- PMLiVE

UCB presentation highlights impacts of epilepsy on daily life

The data underlines the consequences of disrupted sleep for patients and caregivers

- PMLiVE

Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

- PMLiVE

PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

- PMLiVE

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

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

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

- PMLiVE

UCB releases new data on Bimzelx for hidradenitis suppurativa

The disease affects around 1% of the population in most studied countries

- PMLiVE

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

- PMLiVE

AstraZeneca reports positive results from rare disease trial

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

- PMLiVE

UCB’s Fintepla shows positives results for Lennox-Gastaut syndrome

The study demonstrated a sustained reduction in seizures for patients

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