Waldenström’s macroglobulinemia
J&J’s Darzalex combination treatment for untreated multiple myeloma recommended by NICE
Around 33,000 people in the UK are currently living with multiple myeloma, an incurable type of blood cancer
LEO Pharma acquires rights for dersimelagon from Tanabe Pharma
The company will pay $435m in up front and near-term milestone payments
NICE recommends axunio’s Axhidrox cream for adults with severe hyperhydrosis
Excessive sweating affects 1-5% of the population and can have a substantial impact on quality of life
NICE recommends treatments for mantle cell lymphoma and chronic lymphocytic leukaemia
The treatments are for eligible adults in England and Wales
NICE recommends Genmab’s Tivdak for recurrent or metastatic cervical cancer
It is estimated that 3,300 women are diagnosed with cervical cancer in the UK each year
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
Sanofi and Regeneron’s Dupixent receives positive NICE final draft guidance for COPD
Around 1.4 million people in the UK are diagnosed with COPD, with two million thought to be undiagnosed
Havas Life London unveils AI awareness campaign
The campaign highlights the dangers of AI misinformation in rare disease