A group of experts has discussed and released a series of recommendations for future trials’ design in Sanfilippo syndrome. The…
Articles by Patricia Inácio, PhD
Swedish Orphan Biovitrum AB’s (Sobi) has dosed the first patient in its Phase 1/2 clinical trial evaluating SOBI003 to treat Sanfilippo syndrome type…
High numbers of antibodies targeting different types of adeno-associated viruses (AAV) pose challenges for AAV-based gene therapies in Sanfilippo…
A mouse model for Sanfilippo Syndrome type B recreates the main characteristics of humans with the disease, indicating a potential…
Researchers have developed and characterized the first fruit fly model for Sanfilippo syndrome to help advance knowledge about the cellular…
It still takes doctors in Holland up to three years to diagnose the rare disease mucopolysaccharidosis — a situation that…
Mild to moderate hearing loss is common among children and young adults with different mucopolysaccharidosis (MPS) diseases, including Sanfilippo…