Recent Advances To Treat Brain Disorders in Mucopolysaccharidoses Outlined in Review

Several approaches are currently being investigated to allow the delivery of novel therapeutic agents into the central nervous system and target neurological symptoms in patients with mucopolysaccharidosis disorders, including Sanfilippo syndrome. Although patients with mucopolysaccharidosis (MPS) disorders can suffer from neurological disorders that include neurocognitive decline and behavioral problems, treating these…

Lysogene Plans Phase 2/3 Study of LYS-SAF302 Later This Year

Lysogene’s Phase 2/3 trial on LYS-SAF302 gene therapy, a clinical candidate for Sanfilippo syndrome type A, will begin in the second half of 2018 due to manufacturing delays. LYS-SAF302 is a viral vector that carries a healthy SGSH gene. This gene gives instructions to produce an enzyme called sulfamidase, which…

Pentosan Polysulfate Shows Promise in Mouse Study of Sanfilippo Type A

Subcutaneous pentosan polysulfate (PPS) treatment reduces overall inflammation and brain degeneration, improving hyperactivity/anxiety and learning behavior in Sanfillipo syndrome type A, according to a mouse study. The study, “Pentosan Polysulfate Treatment of Mucopolysaccharidosis Type IIIA Mice,” was published in the Journal of Inherited Metabolic Disease.