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activated phosphoinositide 3-kinase delta syndrome

- PMLiVE

LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

- PMLiVE

MHRA authorises Biogen’s higher-dose Spinraza for SMA treatment

SMA is a rare genetic neuromuscular condition affecting around 1,600 people in the UK

- PMLiVE

Shionogi starts phase 2 clinical trial for Pompe Disease

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

Scientist

UK clinical trial regs are changing — here’s what’s happening

Discover the transformative updates to UK clinical trial regulations, effective from April 2026, and learn how they aim to enhance transparency, participant safety, and trial efficiency.

Cuttsy + Cuttsy

- PMLiVE

PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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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

- 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

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

Obesity affects approximately 15 million people in the UK

- PMLiVE

GSK’s Nucala approved by MHRA for uncontrolled COPD

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

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

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