Seelos Therapeutics announced it has received a positive opinion on an orphan drug designation for SLS-005 (trehalose) — its investigational therapy for Sanfilippo syndrome — from the Committee for Orphan Medicinal Products (COMP), a part of the European Medicines Agency (EMA). Following this opinion from…
An ongoing clinical trial is investigating an approved anti-inflammatory compound anakinra, sold under the brand name Kineret, as a potential treatment for Sanfilippo syndrome. The Phase 2/3 pilot trial (NCT04018755) opened in May in the U.S. and has enrolled 20 children, ages 4 and older, all of whom will be…
A multiplex test using a drop of dried blood can be used to screen newborns for eight different lysosomal storage diseases, including Sanfilippo syndrome, a new study shows. The study, “Newborn screening for Morquio disease and other lysosomal storage diseases: results from the 8-plex assay for 70,000…
Injecting BMN 250 — a potential enzyme replacement therapy for Sanflippo type B — directly into the fluid around the brain is a more effective way than intravenous infusion to get the treatment into the brain, a study in animal models suggests. The study, “Translational studies of…
The accumulation of heparan sulfate in Sanfilippo syndrome type B can alter sulfur metabolism in the body, which seems to be sex- and organ-specific, a mouse study suggests. The study, “Effect of glycosaminoglycans accumulation on the non-oxidative sulfur metabolism in mouse model of Sanfilippo syndrome, type…
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