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Press release – NICE condemns to death up to three babies each year in England

NICE the drug decision making body have announced their final decision  today, Wednesday 15 February 2017, not to recommend Kanuma (Sebelipase alfa), the treatment for  individuals suffering from Lysosomal Acid Lipase Deficiency (LAL D). Kanuma was given fast track approval

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FDA permits marketing of first newborn screening system MPS I and Fabry

The U.S. Food and Drug Administration have permitted marketing of the Seeker System for the screening of four, rare Lysosomal Storage Disorders (LSDs) in newborns. The Seeker system is designed to detect Mucopolysaccharidosis Type I (MPS I), Pompe, Gaucher and Fabry.

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Donating with eBay and PayPal – all you need to know

Charity donations with PayPal You can make an instant donation via PayPal to the MPS Society. Just follow these simple steps: 1. Click on this link paypal.com/fundraiser/charity/15394 2. Choose the amount you’d like to give, and whether you’d like to

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Abeona Therapeutics enrolls high dose subject in Sanfilippo clinical trial

ABO-102, the leading clinical gene therapy program for Sanfilippo syndrome type A patients, has demonstrated central nervous system (CNS) and peripheral organ disease biopotency First high dose cohort patient is enrolled, and all patients (n = 4) have cumulative 644

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MPS in numbers

896

individual and families
supported in 2015

25

rare disease supported

3,364,628

£ given research projects
between 1984-2010