MPS awareness in the news


Awareness day 2018

MPS awareness in the news

You’ve been brilliant this MPS Awareness Day spreading the word about these diseases and paying homage to your MPS heroes. As well as a great social media campaign across Twitter, Facebook and Instagram we also reached new audiences through press

idorsia fabry ebook advert

New oral therapy for Fabry patients in development

Idorsia Pharmaceuticals, a Swiss biotech company, is developing lucerastat, a new oral therapy for patients with Fabry Disease. The Phase 3 clinical study MODIFY has just enrolled its first patients and will investigate the use of lucerastat monotherapy for the

Orchard Therapeutics and GSK logo

GSK signs strategic agreement to transfer rare disease gene therapy portfolio to Orchard Therapeutics

Agreement strengthens Orchard’s position as a global leader in gene therapy for rare diseases GSK takes 19.9% equity stake in Orchard and seat on board GSK and Orchard Therapeutics have announced a strategic agreement, under which GSK will transfer its

Abeona Therapeutics

Abeona Therapeutics Appoints Carsten Thiel, Ph.D., as Chief Executive Officer

Five years of growth and progress at Abeona Therapeutics Company strengthens management team with biopharmaceutical veteran bringing extensive commercial experience in life-threatening rare diseases Abeona Therapeutics announces Carsten Thiel, Ph.D., as Chief Executive Officer.  Timothy J. Miller, Ph.D., will remain President

Bob Stevens, Group Chief Executive, MPS Society

Ramblings of a CEO

Why do we do what we do? Well there are many reasons that we work in the rare disease space; money, travel, career, passion, belief that we can make a difference. I recently had some training on health economics and