alpha-1 antitrypsin deficiency
Sanofi announces phase 3 study data for infantile-onset Pompe disease
Pompe disease is a rare, progressive neuromuscular disease, with infantile onset being the most aggressive form
Sanofi’s Cenrifki approved in Europe for secondary progressive MS without relapses
SPMS is a debilitating stage of MS where patients experience increasing levels of disability
Sanofi appoints Paulo Fontoura as Global Head of R&D
Fontoura previously held senior leadership roles at Xaira Therapeutics and Roche
Sanofi’s Sarclisa approved by MHRA as UK’s first anti-cancer treatment given via on-body injector
It is the first anticancer treatment in the UK and globally to be administered this way and a first in multiple myeloma
LEO Pharma to acquire Replay gene therapy platform
The acquisition will advance innovation for patients with rare skin diseases
Sanofi’s Tzield gets expanded FDA approval for early-stage type 1 diabetes
The treatment can be used to delay the onset of clinical type 1 diabetes
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’s Rezurock receives positive CHMP opinion for chronic graft-versus-host disease
Up to 50% of allogeneic haematopoietic stem cell transplant patients develop chronic GVHD
Sanofi and Regeneron’s Dupixent receives positive NICE final draft guidance for COPD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