An experimental enzyme replacement therapy (ERT), called JR-441, for Sanfilippo syndrome type A has been designated an orphan drug by the U.S. Food and Drug Administration, the treatment’s developer, JCR Pharmaceuticals, has announced. The designation is given to therapies being developed for conditions affecting fewer than 200,000 people…
News
Note: This story was updated Dec. 11, 2023, to correct that Kyowa Kirin has entered into an agreement to acquire Orchard Therapeutics, which will be finalized next year. The story also corrected that OTL-200, Libmeldy, is under priority review by the U.S. Food and Drug Administration with a review date…
JCR Pharmaceuticals is now recruiting children and adolescents with Sanfilippo syndrome type A for a Phase 1/2 clinical trial testing its enzyme replacement therapy JR-441 at a single location in Hamburg, Germany. The first patient in the trial recently was dosed, according to a press release from…
A primary goal of this year’s World Sanfilippo Awareness Day, observed annually on Nov. 16, is to get people talking about Sanfilippo syndrome, a rare disorder thought to affect 1 in 70,000 people. “World Sanfilippo Awareness Day is about spreading awareness and sparking conversations globally about Sanfilippo syndrome,…
A carbohydrate molecule derived from marine bacteria was able to reduce inflammation in the brain, reverse metabolic dysfunction, and preserve brain tissue integrity in a mouse model of Sanfilippo syndrome type A, according to a new study. These benefits corresponded to an easing of motor deficits in the mouse…
Standardized tests that measure memory, motor function, and well-being can be used to track the progression of neurological dysfunction in a mouse model of Sanfilippo syndrome type A, a study shows. “Our data show that the water cross maze, hind-limb gait, nest building, and burrowing are useful assessments to…
JCR Pharmaceuticals has won permission from officials in Germany to start a Phase 1/2 clinical trial of JR-441, an experimental therapy designed to deliver to the brain a working version of the enzyme that’s missing in Sanfilippo syndrome type A. The Paul-Ehrlich Institute (PEI), an agency of the…
Researchers are calling for individualized clinical trials that would test the effects of anti-inflammatory therapies for Sanfilippo syndrome and related conditions. A team of scientists from Europe has published a paper that outlines how such trials can be conducted and offered recommendations for specific therapies to test in specific…
Tralesinidase alfa, an experimental enzyme replacement therapy for people with Sanfilippo syndrome type B, was safe and showed good brain distribution in non-human primates, specifically cynomolgus monkeys, a new study found. The treatment was equally well tolerated regardless of dose, duration of the infusion, or formulation. According to…
The National MPS Society will host its Maritime Gala May 13 on the San Francisco Bay in Tiburon, California, at the historic Corinthian Yacht Club to raise funds for its family support and research programs. The event also observes International MPS Awareness Day, observed annually on May…
Recent Posts
- Thinking about my daughter’s future includes planning her funeral
- Enzyme therapy protects kids’ brain function for up to 6 years
- Accepting my daughter’s Sanfilippo diagnosis has allowed me to heal
- Sanfilippo A gene therapy candidate granted access to UK’s ILAP program
- I’m a wife and mom to a special needs child, but what defines me?
- FDA decision on UX111 gene therapy for Sanfilippo expected in September
- Screen time is a lifeline for our daughter with Sanfilippo syndrome
- Physical therapy eases pain, improves life for Sanfilippo kids: Study
- Being a Sanfilippo parent means facing impossible decisions
- UX111 gene therapy shows long-term benefit in Sanfilippo type A: Trial data