Rare Disease Report

Potential Use of Biomarkers in Hurler Disease (MPS I)

FEBRUARY 23, 2013

Brian Bigger, PhD, leads the Stem Cell & Neurotherapies laboratory at the University of Manchester in Manchester U.K.  The lab uses a multidisciplinary approach to investigate stem cell and gene therapies for neurological diseases, including Mucopolysaccharidosis (MPS) diseases such as MPS I (Hurler) and MPS IIIa (Sanfilippo).  In this exclusive interview with Rare Disease Report, Dr. Bigger described the recent  clinical study his lab performed to determine if they could use biomarkers to ascertain best treatment options for MPS I patients. (ie, enzyme replacement therapy vs bone marrow transplant).

The data describe in this video clip was presented by Dr. Bigger’s colleague  Muhammad Saif at the  LDN World Symposium in an oral presentation titled.“ The Majority of MPSI Patients Tested Raise Inhibitory Allo-Antibodies Against Enzyme Replacement Therapy”.

For more information about MPS 1, click here or here. For more information about Dr. Bigger’s research, visit  http://www.human-development.manchester.ac.uk/staff/BrianBigger/

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