Rare Disease

© Getty Images

New treatment for Fabry disease receives key recommendation

By Isabel Cameron

Chiesi, the biopharmaceutical and healthcare group, has announced that the National Institute for Health and Care Excellence (NICE) has recommended Elfabrio (pegunigalsidase alfa) as a treatment for Fabry disease in adults.


Building an mRNA giant: Moderna’s 7 priorities for 2023

By Rachel Arthur

mRNA pioneer Moderna wants to grow and thrive past its initial COVID-19 vaccine success. “To continue to build the best version of Moderna, we have established seven priorities for 2023,” says CEO Stephane Bancel: as the company outlines goals that range...

© GettyImages/niphon

Moderna to develop mRNA therapeutic for very rare disease

By Jane Byrne

Moderna and the nonprofit Institute for Life Changing Medicines (ILCM) are collaborating to develop a new messenger RNA (mRNA) therapeutic (mRNA-3351) for Crigler-Najjar Syndrome Type 1 (CN-1), an ultra-rare disease.