News

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…

An accurate and early diagnosis of Sanfilippo syndrome type C can be difficult, as this subtype is quite rare, but important to prevent life-threatening complications and improve life quality, a case report highlighted. The report, “A Rare case of Sanfilippo syndrome type ‘C’,” and published in the Indian…

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…

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,…

Problems with one or more senses are common in people with Sanfilippo syndrome and other mucopolysaccharidoses (MPSs), but there is very little research into how these impairments affect daily living or life quality, a new paper indicates. Collecting data using questionnaires or forms filled in by patients that…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

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…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

The European Medicines Agency has granted orphan drug designation to SLS-005 (trehalose) for the treatment of Sanfilippo syndrome. This designation, which follows a positive opinion from the agency’s Committee for Orphan Medicinal Products, was announced in a press release from Seelos…