Early data from the AFFINITY DUCHENNE trial of a new gene therapy, RGX-202, raise hopes for a breakthrough in Duchenne muscular dystrophy (DMD). 6 March 2024
The National Institute for Health and Care Excellence (NICE) has decided not to recommend Xenpozyme (olipudase alfa) as a treatment for acid sphingomyelinase de 5 March 2024
Drugmakers, patient advocate groups, policymakers and others have come together for Rare Disease Day, which took place at the end of February, to both celebrate 4 March 2024
Last month, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended the refusal of the marketing authorization f 29 February 2024
A commercial stage biopharmaceutical R&D company, focused on the development of products that address the medical challenges in the therapeutic area of anti-infectives.