Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases

Amicus Therapeutics has opened its Global Research and Gene Therapy Center of Excellence that will seek to advance research programs in Rett Syndrome and several other rare diseases. Besides Rett, the Center will focus on Angelman Syndrome, muscular dystrophies and lysosomal storage disorders, such as Pompe and…

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…