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Latest news

Lysogene launches a prospective, observational study of MPS IIIA

Lysogene has launched a prospective, observational study of patients, who are aged nine years old or younger, with mucopolysaccharidosis IIIA (MPS IIIA) to better understand their health problems and how to measure these problems over time. This is an observational ...

Approval for Galafold™ in patients with Fabry announced

Amicus Therapeutics, a biotechnology company at the forefront of rare and orphan diseases, today announced that the European Commission has granted full approval for the oral small molecule pharmacological chaperone Galafold™ (migalastat) as a first line therapy for long-term treatment of adults and ...

Thank you!

Thank you to everyone who helped us to mark MPS Awareness Day on 15th May 2016!

Take to the Skies for MPS Awareness Day!

We are delighted to have teamed up with Shire, the National MPS Society and the International MPS Network to bring you a brand new campaign to mark MPS Awareness Day!

MPS in numbers