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…
News
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…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
Seelos Therapeutics announced it has received a positive opinion on an orphan drug designation for SLS-005 (trehalose) — its investigational therapy for Sanfilippo syndrome — from the Committee for Orphan Medicinal Products (COMP), a part of the European Medicines Agency (EMA). Following this opinion from…
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience. Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…
Recent Posts
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- My son is starting to ask questions about his special needs sibling
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