thymidine kinase 2 deficiency
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 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
UCB presentation highlights impacts of epilepsy on daily life
The data underlines the consequences of disrupted sleep for patients and caregivers
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
UCB releases new data on Bimzelx for hidradenitis suppurativa
The disease affects around 1% of the population in most studied countries
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
AstraZeneca announces $2bn investment in US manufacturing
The new and expanded facilities will support production of rare disease drug treatments