Latest News

image

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

Read More
image

MPS I Clinical trial update ArmaGen AGT-181: Investigational Therapy for the Treatment of Patients with Hurler Syndrome

Following ArmaGen’s data presentation at WORLD Symposium they have developed a Clinical Trial Update that provides an overview of the data on AGT-181 therapy. The full press release is available below. ArmaGen’s AGT-181 Demonstrates Neurocognitive Benefit in Children with Severe

Read More
image

Ramblings of a CEO Part 1

Our new Chief Executive has been reporting back on his experiences and what he is learning at the 14th Annual WORLDSymposium. So what have I learned so far from the WORLD gathering? 1. The passion to make a difference here

Read More
image

Protalix BioTherapeutics’ pegunigalsidase alfa Receives Fast Track Designation from the U.S. Food and Drug Administration

Fast Track designation highlights high unmet medical need in the treatment of Fabry disease Protalix BioTherapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to pegunigalsidase alfa, or PRX-102, the Company’s plant cell-expressed recombinant,

Read More

MPS in numbers

896

individual and families
supported in 2015

25

rare disease supported

3,364,628

£ given research projects
between 1984-2010