The case of a 44-year-old woman who was diagnosed with Sanfilippo syndrome type A co-occurring with a heart abnormality…
Articles by Marisa Wexler, MS
The U.S. Food and Drug Administration (FDA) is requesting changes to Ultragenyx Pharmaceutical’s manufacturing process before it will consider…
Ultragenyx Pharmaceutical’s experimental gene therapy UX111 slows or halts the brain damage that affects communication and cognitive and motor…
GC1130A, an experimental enzyme replacement therapy (ERT) for Sanfilippo syndrome type A, has been put on the fast track…
Over the next two decades, the economic burden of Sanfilippo syndrome in the U.S. is expected to exceed $2…
Most children with Sanfilippo syndrome type A who received the one-time gene therapy UX111 early in life in a clinical…
Standardized tests that measure memory, motor function, and well-being can be used to track the progression of neurological dysfunction in…
JCR Pharmaceuticals has won permission from officials in Germany to start a Phase 1/2 clinical trial of JR-441, an…
Researchers are calling for individualized clinical trials that would test the effects of anti-inflammatory therapies for Sanfilippo syndrome and…
Light-sensing cells in the eyes become damaged and die in Sanfilippo syndrome type C before the onset of behavioral…