Mary Chapman,  features writer—

An emotional video about a boy with Sanfilippo syndrome and his parents’ fight to save him has raised about $244,000 toward a first-ever clinical trial that could give the youngster and others a chance at life. The two-and-a-half-minute “Save Connor” video, which has “gone viral,” raised $100,000 in…

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…

The genetic disorder Sanfilippo syndrome, also known as mucopolysaccharidosis type III, is so rare that most physicians are unfamiliar with it. So, it is not surprising that it may have taken years for your child to receive an accurate diagnosis. Learning the cause of your child’s symptoms often puts you on a…

Sanfilippo syndrome is a neurodegenerative disorder that affects about 1 in 70,000 births. Raising awareness of this rare genetic disease could mean that more patients would benefit from earlier diagnosis and treatment. Here are some facts about Sanfilippo syndrome, and efforts to make it more widely…

On Nov. 16, World Sanfilippo Awareness Day, supporters will bring global attention to the rare neurodegenerative disorder, hopefully sparking conversations globally about the community and its needs. From sharing images on social media and using a special Facebook photo frame, to displaying the official Awareness Day logo and participating…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

A new initiative led by the Cure Sanfilippo Foundation is seeking to provide more insight on the impact of remote learning on children with Sanfilippo syndrome and other developmental disorders. The advocacy effort is being spearheaded by the organization’s chief science officer and co-founder, Cara O’Neill. The goal…

Eating and drinking can be problematic in Sanfilippo syndrome, a rare neurodegenerative disorder characterized by intellectual and behavioral disabilities. Finding out your child may need a feeding tube can be overwhelming, particularly when other medical issues are present. Here’s some information that may help. What is Sanfilippo syndrome? Sanfilippo syndrome…

Sanfilippo syndrome patients and caregivers are being asked to help speed the development of potential disease treatments by joining  AllStripes, and contributing their de-identified medical records to research efforts. The San Francisco-based company, previously known as RDMD, uses its rare disease research platform to aggregate and analyze such information,…

The Canadian Glycomics Network (GlycoNet) has granted $3.9 million in funding for research projects aimed at mitigating the healthcare burden in Canada and contributing to a better understanding of diseases, including Sanfilippo syndrome. The recipients — multidisciplinary research institutions and their industry partners — will use the funding…