Many efforts have been made to improve care and find a cure for patients with Sanfilippo disease. While some pursuits…
Articles by Alice Melão
The U.S. Food and Drug Administration (FDA) has accepted an investigational new drug (IND) application for Swedish Orphan Biovitrum AB‘s…
Heparan sulfate accumulation is not a good indicator of Sanfilippo syndrome type A symptom onset, a new mouse model…