Margarida Maia, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has selected DNL126, an enzyme replacement therapy being developed by Denali Therapeutics for Sanfilippo syndrome type A, to join its START pilot program — fully, Support for Clinical Trials Advancing Rare Disease Therapeutics. As part of this pilot program, launched last…

GC Biopharma and Novel Pharma plan to initiate a Phase 1 clinical trial this year to test the safety and tolerability of  GC1130A, an investigational enzyme replacement therapy that the companies are developing for Sanfilippo syndrome type A. The announcement follows the U.S. Food and Drug Administration…

The European Medicines Agency (EMA) has granted orphan drug status to GC1130A, an investigational brain-penetrant enzyme replacement therapy that’s in preclinical testing for Sanfilippo syndrome type A. GC1130A, which is being developed by GC Biopharma with Novel Pharma, was also awarded…

JCR Pharmaceuticals is now recruiting children and adolescents with Sanfilippo syndrome type A for a Phase 1/2 clinical trial testing its enzyme replacement therapy JR-441 at a single location in Hamburg, Germany. The first patient in the trial recently was dosed, according to a press release from…

Four out of the five children with Sanfilippo syndrome type A in a Phase 1/2 clinical trial have continued to gain cognitive skills — which in three were similar to healthy children — after being given OTL-201, an experimental gene therapy being developed by Orchard Therapeutics. The children…

The Cure Sanfilippo Foundation continues to support an expanded newborn screening program that includes testing for Sanfilippo syndrome, now by signing on to a letter that the EveryLife Foundation is submitting to legislators in the U.S. states of Maryland and Mississippi. EveryLife‘s letter was written to thank…

Properties of a lubricant of the small air sacs (alveoli) that is essential for normal lung function appear to be altered in Sanfilippo syndrome type A, a study in mice reported. Changes in the mechanism and activity of this lung lubricant, known as pulmonary surfactant, “may explain the increased susceptibility of…

Sanfilippo carriers likely are not at an increased risk of developing adult-onset dementia or other neurodegenerative conditions, despite earlier clinical evidence suggesting such a predisposition, a new study in mice has found. After examining the brains of laboratory mice, the investigators concluded that their mouse model “does not exhibit…