activated phosphoinositide 3-kinase delta syndrome
Ogsiveo recommended by NICE to treat rare soft tissue tumours
Desmoid tumours are rare soft tissue tumours that can grow aggressively into nearby tissues
MHRA approves Pharma Mar’s lurbinectedin for extensive-stage small cell lung cancer
The treatment is for adults in combination with atezolizumab
Moderna’s updated COVID-19 mRNA vaccine approved by MHRA
The updated vaccine will be available to eligible groups as part of the 2026/27 NHS vaccination programme
LEO Pharma acquires rights for dersimelagon from Tanabe Pharma
The company will pay $435m in up front and near-term milestone payments
Eli Lilly’s Foundayo authorised by UK MHRA for weight loss and type 2 diabetes
The UK is the first country in Europe to authorise the oral medication
LEO Pharma to acquire Replay gene therapy platform
The acquisition will advance innovation for patients with rare skin diseases
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
Shionogi starts phase 2 clinical trial for Pompe Disease
The rare disease affects around one in every 22,000 people worldwide
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.
PharmaQuest returns: EPA perspectives, through a rare disease lens
By Lottie Sequerra and Emily Morton-West
MHRA approves Leqselvi for severe alopecia areata
Around 30% of patients saw 80% more scalp hair after 24 weeks of treatment
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