Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The…
Articles by Mary Chapman
The Cure Sanfilippo Foundation will be among those presenting posters at the WORLDSymposium 2021 conference on lysosomal storage disorders…
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “…
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and…
There may come a time when you will need to consider palliative care for your child with Sanfilippo syndrome, and…
Sanfilippo syndrome is a neurodegenerative disorder that affects about one in 70,000 births. Because it’s rare, many physicians are unfamiliar…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to…
Clinical trials are an essential step in treatment development, yet the many people they could potentially help are either…
If your child has been diagnosed with Sanfilippo syndrome, you may be feeling mixed emotions, from shock to concern to…
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients…