Pentosan Polysulfate May Reduce Inflammation in Gaucher, Fabry, Study Suggests

Pentosan polysulfate (PPS), an approved medicine, may be a potential add-on treatment for Gaucher disease, helping reduce inflammation and bone alterations in cellular models of the condition, a study found. The treatment was also effective in cell models of Fabry disease, another lysosomal storage disorder. The study, “…

Approved Bladder Pain Therapy Being Repurposed for Lysosomal Storage Disorders

The Icahn School of Medicine at Mount Sinai is repurposing pentosan polysulfate sodium — an anti-inflammatory medicine approved as Elmiron for bladder pain — for the treatment of lysosomal storage disorders, possibly to include Gaucher disease. The medical school in New York has signed two licensing agreements with Australia-based Paradigm…