Nature

High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector

Mucopolysaccharidosis type IIIA (MPS-IIIA) or Sanfilippo A syndrome is a lysosomal storage genetic disease that results from the deficiency of the N-sulfoglucosamine sulfohydrolase (SGSH) protein, a ...
University of Manchester

Early results of gene therapy trial for ‘childhood dementia’ show promise

Researchers will tell an international conference today (24/02/23) that an investigational gene therapy for Sanfilippo syndrome - which leads to a form of childhood dementia - has shown promising ...
Seeking Alpha

Denali Therapeutics Announces FDA Has Selected DNL126 (ETV:SGSH) for MPS IIIA (Sanfilippo Syndrome Type A) for START Pilot Program Intended to Accelerate Development of Rare ...

START is a pilot program newly launched jointly by the FDA CDER and CBER divisions to further accelerate the development of novel drug and biological products for rare diseases DNL126 was selected as ...
Nasdaq

Ultragenyx Pharmaceutical Inc. Receives Complete Response Letter from FDA for UX111 Gene Therapy for Sanfilippo Syndrome Type A

Ultragenyx received a Complete Response Letter from the FDA regarding its UX111 therapy for Sanfilippo syndrome type A, citing manufacturing observations. Ultragenyx Pharmaceutical Inc. announced that ...