Who do you know who has shown courage over adversity?



Who do you know who has shown courage over adversity?

MPS Society anniversary awards May 2017 is the 35th Anniversary of the founding of the Society for Mucopolysaccharide Diseases. To celebrate this milestone the Board of Trustees will at the MPS Gala Dinner at the conference on Saturday 8 July

Ultragenyx announces investigational therapy for MPS VII accepted for review

Ultragenyx Announces Recombinant Human Beta-Glucuronidase Biologics License Application and Marketing Authorization Application Filed and Accepted for Review; FDA Grants Priority Review Status Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases,

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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

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

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