Forest Ray PhD,  —

Parents of children with Sanfilippo syndrome place high value on therapies that provide even modest benefits to a narrower set of symptoms than most clinical trials address, according to a recent study. Understanding the impacts felt by caregivers and their treatment priorities can help determine the outcomes measured in…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

The Sanfilippo Children’s Foundation, in Australia, has awarded AU$355,000 (about USD$267,000) for a two-year project to determine whether medicines targeting the immune system could be used to treat children with Sanfilippo syndrome. Researchers will use the grant to screen medicinal compounds for their ability to prevent cell degeneration…

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…

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 University of Washington and the National MPS Society are seeking  participants for a study to predict the type and severity of MPS (mucopolysaccharidosis) disorders, such as Sanfilippo syndrome (MPS III). Results of the study will be used to improve newborn screening efforts. Participants with MPS II (Hunter syndrome),…

The Committee for Orphan Medicinal Products, a branch of the European Medicines Agency (EMA), favors designating SLS-005 (trehalose) an orphan drug as a potential treatment for Sanfilippo syndrome, Seelos Therapeutics, its developer, announced. The positive opinion will now be sent to the European Commission, which…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

The U.S. Food and Drug Administration (FDA) has issued a hold on Lysogene‘s trial of its gene therapy candidate LYS-SAF302 for Sanfilippo syndrome type A. LYS-SAF302 is a one-time treatment meant to deliver a healthy copy of the SGSH gene, which is defective in Sanfilippo…