Latest News

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Abeona Therapeutics Receives Regulatory Approval to Initiate Clinical Trial in Australia with ABO-102 Gene Therapy For Patients with MPS IIIA

Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, has announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type

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MPS Awareness Day is just round the corner

It’s MPS Awareness Day on Monday 15 May and there’s still time to boost awareness on this special day. Here’s our FAQ guide to all the ways you can get involved.   Can I donate something other than money? Yes! You can donate

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An update on the BioMarin MPS IIIB clinical development programme

An update for patient associations and families from BioMarin April 2017 BioMarin have received a number of enquiries regarding the status of their clinical development program in Sanfilippo Syndrome Type B (MPS IIIB). The program, which involves multiple centres around

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Join us to celebrate MPS Awareness Day at Gulliver’s Land, Milton Keynes

To celebrate MPS Awareness Day 2017 we are offering lucky families the opportunity to visit Gulliver’s Theme Park in Milton Keynes on Sunday 14th May 2017. If you’re looking for a family fun-filled day out for you and your little ones

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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