Seelos Therapeutics announced it has received a positive opinion on an orphan drug designation for SLS-005 (trehalose)…
Articles by Marisa Horak, MS
An ongoing clinical trial is investigating an approved anti-inflammatory compound anakinra, sold under the brand name Kineret, as a potential treatment for…
A multiplex test using a drop of dried blood can be used to screen newborns for eight different lysosomal storage…
Injecting BMN 250 — a potential enzyme replacement therapy for Sanflippo type B — directly into the fluid around the…
The accumulation of heparan sulfate in Sanfilippo syndrome type B can alter sulfur metabolism in the body, which seems…