Spinal cord decompression — a type of spinal surgery — should be done on mucopolysaccharidosis (MPS) patients who have…
Articles by Iqra Mumal, MSc
Sarepta Therapeutics has signed a licensing agreement with Lysogene for the development of investigational gene therapy LYS-SAF302 to…
Results from a Phase 3 clinical trial evaluating the use of high-dose genistein in children with Sanfilippo syndrome shows…
Brain transplantation of genetically corrected stem cells partially restored enzymatic activity in mice with Sanfilippo syndrome, according to UCLA researchers.
An investigative gene therapy to treat patients with Sanfilippo syndrome type C was developed at the University of Manchester…
A patient with Sanfilippo syndrome and poor nutritional status developed high levels of homocysteine —  an amino acid likely due…
Injecting a modified version of the NAGLU enzyme directly into the brains of mice with Sanfilippo syndrome type B…
Abeona Therapeutics announced that ABO-102 (AAV-SGSH), an investigational therapy for Sanfilippo syndrome type A (MPS IIIA), demonstrated significant and…