Spruce Biosciences said it plans to launch a Phase 3 clinical study of tralesinidase alfa as an enzyme replacement therapy…
Articles by Margarida Maia, PhD
The U.S. Food and Drug Administration (FDA) has selected DNL126, an enzyme replacement therapy being developed by Denali Therapeutics…
GC Biopharma and Novel Pharma plan to initiate a Phase 1 clinical trial this year to test the…
The European Medicines Agency (EMA) has granted orphan drug status to GC1130A, an investigational brain-penetrant enzyme replacement therapy…
JCR Pharmaceuticals is now recruiting children and adolescents with Sanfilippo syndrome type A for a Phase 1/2 clinical…
Four out of the five children with Sanfilippo syndrome type A in a Phase 1/2 clinical trial have continued…
The Cure Sanfilippo Foundation continues to support an expanded newborn screening program that includes testing for Sanfilippo syndrome,…
Properties of a lubricant of the small air sacs (alveoli) that is essential for normal lung function appear to be…
Sanfilippo carriers likely are not at an increased risk of developing adult-onset dementia or other neurodegenerative conditions, despite earlier…