Enrollment is now open for SanfilippoLINK, a global clinical research and patient registry platform that aims to advance scientific understanding of Sanfilippo syndrome. The platform, operated by the Cure Sanfilippo Foundation, is open to people with all types of Sanfilippo syndrome. The platform consists…
News
Long-term treatment with the investigational enzyme replacement therapy tralesinidase alfa stabilized cognitive function for up to six years in children with Sanfilippo syndrome type B, clinical trial data showed. Spruce Biosciences, the therapy’s developer, plans to use the data to support an application seeking accelerated approval of the…
Regulatory authorities in the U.K. have granted innovation passport designation to OTL-201, an experimental gene therapy for Sanfilippo syndrome type A, generally the disease’s most severe form. This new status gives treatment developer Orchard Therapeutics (recently acquired by Kyowa Kirin) access to a U.K. program…
The U.S. Food and Drug Administration (FDA) is once again considering whether or not it will approve UX111, Ultragenyx Pharmaceutical’s experimental gene therapy for Sanfilippo syndrome type A. According to Ultragenyx, the FDA has accepted a resubmitted biologics license application (BLA), and a decision from the agency is expected…
Physical therapy (PT) appears to be safe for children with Sanfilippo syndrome and other types of mucopolysaccharidosis (MPS), and may help maintain function, ease pain, and support quality of life, according to a small study. However, the researchers said, many families were unable to attend all scheduled therapy sessions, underscoring…
A single dose of UX111 (rebisufligene etisparvovec), an experimental gene therapy being developed by Ultragenyx Pharmaceutical, reduces the toxic buildup of heparan sulfate for more than eight years and helps children with Sanfilippo syndrome type A maintain or improve developmental skills, particularly when administered early. These long-term results…
The total economic burden of Sanfilippo syndrome in the U.S. is on track to exceed $2 billion in the next 20 years, according to a new analysis. The figure accounts for the direct costs of managing the condition, along with lost productivity for patients and their families. While Sanfilippo…
A bone marrow transplant may help improve motor function in children with Sanfilippo syndrome, according to a new study from China in which one youngster with the rare genetic disorder had better hand-eye coordination after undergoing the procedure. While the study overall involved dozens of children with similar genetic…
Delivering a lab-made version of the missing enzyme in Sanfilippo syndrome type D directly into the brain reduced toxic heparan sulfate buildup, a key Sanfilippo event, and eased signs of inflammation in a mouse model of the disease, a new study reports. These benefits were observed even when the…
Tralesinidase alfa, also known as TA-ERT, Spruce Biosciences’ investigational enzyme replacement therapy (ERT) for children with Sanfilippo syndrome type B, has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA). The designation is intended to speed up the development and review of treatments that show early evidence…
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