rare diseases action plan for England
Alexion and Aide Health launch app for people with rare diseases
More than 3.5 million people in the UK live with a rare condition
Shionogi to acquire IntraBio in deal worth $2bn
The deal will expand Shionogi’s growing portfolio with IntraBio’s rare diseases capabilities
Ogsiveo recommended by NICE to treat rare soft tissue tumours
Desmoid tumours are rare soft tissue tumours that can grow aggressively into nearby tissues
CSL Seqirus announces strengthened vaccine partnership with UK government
The partnership introduces a world-first flu vaccine and extends the health security pandemic agreement
LEO Pharma acquires rights for dersimelagon from Tanabe Pharma
The company will pay $435m in up front and near-term milestone payments
LEO Pharma to acquire Replay gene therapy platform
The acquisition will advance innovation for patients with rare skin diseases
Shionogi starts phase 2 clinical trial for Pompe Disease
The rare disease affects around one in every 22,000 people worldwide
PharmaQuest returns: EPA perspectives, through a rare disease lens
By Lottie Sequerra and Emily Morton-West
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
Sanofi announces positive results for venglustat in type 3 Gaucher disease
There are currently no approved treatments for the neurological symptoms of this disease
Havas Life London unveils AI awareness campaign
The campaign highlights the dangers of AI misinformation in rare disease
FDA approves first gene therapy for Wiskott-Aldrich syndrome
Symptoms of the rare disease include bleeding, eczema and frequent infections