There are currently four types of Sanfilippo syndrome, Type A, B, C and D, which are caused by a lack of enzymes, which helps to break down and recycle long chain sugars and results in a build-up of ...

A local Natchez family’s lives changed forever on April 4 of this year when their daughter was diagnosed with a rare genetic ...

About Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) Mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A, is an autosomal recessive disease caused by pathogenic mutations ...

Ltd. (TSE 4552; "JCR") announced the initiation of the first patient dosing in Japan in the Phase I clinical trial of JR-441, an investigational enzyme replacement therapy for the treatment of ...

based gene therapy for Sanfilippo syndrome type A (MPS IIIA), and ABO-101 (AAV NAGLU), an AAV based gene therapy for Sanfilippo syndrome type B (MPS IIIB). The company was founded in 1974 and is ...

Soon after, Logan was diagnosed with Sanfilippo syndrome. As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a terminal, neurodegenerative rare disease. Children lose all the skills ...

Good afternoon and welcome to the Ultragenyx third-quarter 2024 financial results conference call. (Operator Instructions) It is now my pleasure to turn the call to Joshua Higa, Vice President of ...

FDA accepts BLA resubmission of pz-cel in recessive dystrophic epidermolysis bullosa and sets PDUFA target action date of April 29, 2025Company ...