dystrophic epidermolysis bullosa
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
LEO Pharma elects Henriette Mersebach to the Board of Directors
Mersebach previously held senior leadership positions at ALK-Abelló and Novo Nordisk
LEO Pharma acquires rights for dersimelagon from Tanabe Pharma
The company will pay $435m in up front and near-term milestone payments
LEO Pharma and Allergy UK launch ‘Don’t Ignore’ chronic hand eczema campaign
The campaign aims to improve awareness and understanding of the condition
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
Matt Ferriday announced as General Manager for LEO Pharma UK
Ferriday most recently held senior leadership roles at Ipsen Biopharma
PharmaQuest returns: EPA perspectives, through a rare disease lens
By Lottie Sequerra and Emily Morton-West
Almirall celebrates new offices in China to advance innovation in dermatology
The company’s R&D and commercial expertise represent valuable capabilities for future Chinese partners
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