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…
News
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…
An adolescent boy was diagnosed with Sanfilippo syndrome type A after the onset of an acute and transient psychotic disorder, according to a case study. The boy’s recurrent psychotic episodes, marked by rapid behavioral changes, were successfully treated with anti-psychotic medications. “This study emphasizes the need for [metabolic] evaluation in…
The case of a 44-year-old woman who was diagnosed with Sanfilippo syndrome type A co-occurring with a heart abnormality called sick sinus syndrome (SSS) was described in a recent report. This is the first documented case of SSS in someone with Sanfilippo syndrome, according to researchers. The study, “…
The U.S. Food and Drug Administration (FDA) is requesting changes to Ultragenyx Pharmaceutical’s manufacturing process before it will consider accelerated approval of UX111, the company’s experimental gene therapy for Sanfilippo syndrome type A. In a corporate update earlier this week, Ultragenyx stated it “believes the observations are readily…
The European Commission has granted orphan drug status to JR-446, a brain-penetrating enzyme replacement therapy (ERT) for Sanfilippo syndrome type B. The designation, announced in a press release from developer JCR Pharmaceuticals, is aimed at accelerating the clinical development and review of investigational therapies that have the…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to JR-446, JCR Pharmaceuticals’ brain-penetrating experimental enzyme replacement therapy (ERT) for Sanfilippo syndrome type B, the company has announced. The designation is meant to accelerate the clinical development and review of investigational therapies with the potential…
Spruce Biosciences said it plans to launch a Phase 3 clinical study of tralesinidase alfa as an enzyme replacement therapy for children with Sanfilippo syndrome type B, a move toward seeking accelerated approval of the treatment. The company said it plans to file a biologics license application (BLA) in…
A hematopoietic stem and progenitor cell (HSPC) transplant eased several disease symptoms, including motor problems and enlarged organs, in a new mouse model of Sanfilippo syndrome type C, according to a study. HSPC transplant provides blood cell precursors from a healthy donor, which are then able to develop into…
The U.S. Food and Drug Administration (FDA) has accepted Ultragenyx Pharmaceutical‘s application seeking approval of UX111, a gene therapy for Sanfilippo syndrome type A, and granted it priority review, which will speed the agency’s decision. By being granted priority review, the biologics license application (BLA) will be…
Recent Posts
- The support system that allows my special needs child to thrive
- Enzyme therapy directly into the brain tested for Sanfilippo type D
- I’m finally seeing a therapist, 3 years after my daughter’s diagnosis
- Tralesinidase alfa ERT for Sanfilippo type B children put on fast track
- Educating others about Sanfilippo syndrome, one bite at a time
- Teen’s acute psychotic disorder leads to Sanfilippo A diagnosis
- Feeding our toddler with Sanfilippo syndrome can be stressful
- It’s hard to stay positive when Sanfilippo is so cruel
- Heart condition SSS found in Sanfilippo patient for first time
- Outside, the world is pressing forward; inside, progress is still