A new study shows a 30-minute, inexpensive, and easy-to-use test may help measure the activity of NDST1, one of the main enzymes involved in heparan sulfate, the sugar molecule that build ups and causes Sanfilippo syndrome. Sanfilippo syndrome, or MPS III, is a...
We are disappointed to hear that Abeona Therapeutics has decided to end enrollment for their clinical trial (Study ABT-003) for MPS IIIA children aged 2-18 years with a Developmental Quotient score less than 60. The company has recently conducted a review of...
Courageous Parents Network is a non-profit organization and educational platform that orients, empowers and accompanies families and providers caring for children with serious illness. They have videos, podcasts, printable guides, Guided Pathways, and a blog to help...
This will be held on the evening of Saturday, July 22, 2023 in conjunction with the National Family Conference. Head to our National Family Conference page for more details!
This popular event takes place every 2 years. This year’s is scheduled for July 21 – 23, 2023 in Calgary Alberta. See our National Conference page for more...
