Latest News

image

Update on the BioMarin Sanfilippo Syndrome Type B (MPS IIIB) Clinical Development Program

An Update for Patient Associations and Families from BioMarin May 2018 We would like to update you about the status of our clinical development program in Sanfilippo B. The BioMarin program, which involves multiple centers around the world, consists of

Read More
image

Stand on your own – Adult MPS patient meeting in Germany

This fellowship has now closed and is not accepting any more applications. Adult  MPS  patients  meeting STAND  ON  YOUR  OWN October  4  and  5,  2018 Maritim  Hotel  Gelsenkirchen,  Germany  For  the  first  time  MPS  Europe  is  organising  Adult  MPS  patients

Read More
image

Sangamo Announces Treatment of First Patient in Phase 1/2 Clinical Trial Evaluating SB-318 Investigational in Genome Editing Therapy for MPS I

The EMPOWERS Study is an open-label, ascending dose clinical trial designed to assess the safety, tolerability and preliminary efficacy of SB-318 in up to nine adult subjects with attenuated MPS I. The study is currently screening subjects at hospitals specializing

Read More
image

Press release on stem cell gene therapy approach for MPSII Hunter disease

A team at The University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients with the disease. Although there

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