High dose genistein treatment in children with Sanfilippo disease shows no clinically meaningful benefit A Phase III, Double Blinded, Randomised, Placebo Controlled Clinical Trial of High Dose Oral Genistein Aglycone in Patients with Sanfilippo Syndrome (Mucopolysaccharidosis III A, B and
Meeting in San Diego at the 15th International Symposium on MPS and related diseases, over 20 of the 40 patient organisations that form the International MPS Network (IMPSN) came together to breathe new life into the group. The aim of
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
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
896
individual and families
supported in 2015
25
rare disease supported
3,364,628
£ given research projects
between 1984-2010