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Gain Morquio B Bone Replacement Equipment & Supplies
1 equipment items found
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
These mutations result in the misfolding and subsequent dysfunction of GLB, which leads to the toxic substrate accumulation of keratan sulfate in organs and tissues. Very limited and investigational symptomatic treatment options include substrate reduction therapy, enzyme replacement therapy, bone marrow transplantation, stem cell transplantation and gene therapy ...