Sanfilippo syndrome, also known as mucopolysaccharidosis type III, is a rare life-threatening disorder that interferes with metabolism. While it doesn’t have a cure, some symptoms can be treated ...

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a neurodegenerative disease in which the body is missing, or does not have enough of, certain enzymes needed to ...

The US Food and Drug Administration (FDA) on Tuesday issued draft guidance making recommendations to drugmakers looking to develop products to treat the rare disease mucopolysaccharidosis type III ...

The syndrome-specific recognition rates ranged from 0% (mucopolysaccharidosis type III) to 83% (Cornelia de Lange syndrome). For detailed results, see Figure 3. The overall recognition rate (58% ...

Jan. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II ...

Sanfilippo syndrome, also called mucopolysaccharidosis (MPS) type III, is a genetically inherited metabolic disorder that causes nervous system issues and other symptoms in children. This syndrome ...

Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a rare neurodegenerative disease that first appears in early childhood. There are four subtypes of Sanfilippo syndrome ...