Rare diseases

News
Alnylam

Alnylam rare disease drug may be delayed

Alnylam may have to put hold off plans for an early FDA approval for its gene-silencing drug givosiran – because recruitment to a rare disease trial is going better than expected.<

Views & Analysis
RareFest_feature_image-20_09_2018

Raising awareness about rare diseases

A family tragedy has prompted Kay Parkinson, founder of the rare disease charity Alstrom Syndrome UK, to create a conference encouraging joined-up thinking to help improve treatments, care and serv