SOBI003, Swedish Orphan Biovitrum AB (Sobi)’s chemically modified version of sulfamidase — the missing enzyme in Sanfilippo…
Articles by Marta Figueiredo, PhD
Researchers have developed two induced pluripotent stem cell (iPSC) lines with NAGLU mutations — the underlying cause of…
Lysogene’s gene therapy candidate LYS-SAF302 corrected enzymatic deficiency, normalized heparan sulfate levels, and reduced brain lesions and neuroinflammation…
Gene therapy has the potential to effectively target the underlying cause of Sanfilippo syndrome and delay or prevent neurodegeneration,…
Allievex, a new clinical-stage biotechnology company founded by Pappas Capital, will continue the clinical program of BioMarin…
Growth impairment among mucopolysaccharidosis (MPS) disorders is milder in people with MPS III (Sanfilippo syndrome) and most…
Sanfilippo syndrome type A leads to changes in the metabolism of gangliosides (sugar-fat molecules) and sphingolipids (fat molecules)…
Sanfilippo syndrome type A leads to toxic accumulation of several molecules in nerve cell axons — the structures responsible for…
While toxic accumulation of large sugar molecules, called heparan sulfate, are present in certain mucopolysaccharidosis (MPS) disorders with brain involvement,…
Sanfilippo syndrome type A leads to the accumulation of other large sugar molecules besides the characteristic heparan sulfate in several…