Heart problems are less common, and milder in severity, in people with Sanfilippo syndrome compared with other forms of…
Articles by Alice Melão
Active tissue remodeling and treatment-resistant accumulation of toxic molecules are contributing factors in the development of persistent upper airway disease…
Alpha-synuclein protein is not responsible for the onset of cognitive and motor symptoms associated with Sanfilippo syndrome type A,…
Nonprofit research foundation Team Sanfilippo (TSF) is preparing to launch a Phase 2 clinical trial to explore the safety…
Abeona Therapeutics is planning to expand its ongoing ABO-102 gene therapy Phase 1/2 trial to include patients with Sanfilippo…
Two new experimental stem cell lines derived from a patient with mucopolysaccharydosis IIIB (MPSIIIB), also known as Sanfilippo syndrome…
Administration of an engineered version of the heparan-N-sulfatase (HNS) enzyme into the cerebrospinal fluid directly through the spinal canal failed to…
Very little is still known about the clinical features and natural course of Sanfilippo syndrome, also known as…
A comprehensive analysis of compounds present in the urine of patients with Sanfilippo syndrome provides new insight into the biological processes…
A common sugar often used as a food additive was seen to ease inflammation in the brain and retina, and…