European Court of Justice
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
AstraZeneca announces $2bn investment in US manufacturing
The new and expanded facilities will support production of rare disease drug treatments
AstraZeneca reports positive results from rare disease trial
HES is a group of rare disorders characterised by persistently elevated levels of white blood cells
Amgen’s Uplizna approved by FDA as first treatment for rare disease IgG4-RD
Immunoglobulin G4-related disease affects approximately 20,000 people in the US
FDA approves Soleno’s Vykat XR as first treatment for hyperphagia in Prader-Willi syndrome
The rare genetic disorder affects an estimated one in every 15,000 newborns in the US
Novartis’ Fabhalta granted FDA approval to treat ultra-rare kidney disease C3G
Approximately half of C3G patients progress to kidney failure within ten years of diagnosis