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Ultragenyx Takes Over Gene Therapy For Sanfilippo Syndrome Type A (MPS IIIA) 

Ultragenyx Takes Over Gene Therapy For Sanfilippo Syndrome Type A (MPS IIIA) 

by adminmps | Sep 6, 2022 | Clinical Trials, News, Research

In our March edition we reported that Abeona Therapeutics had decided to end enrollment for their clinical trial for MPS IIIA children. We are now happy to share that Ultragenyx will be taking over the clinical trials for AAV gene therapy ABO-102 (now UX111). “Based...
Ultragenyx Takes Over Gene Therapy For Sanfilippo Syndrome Type A (MPS IIIA) 

Another Potential Gene Therapy to Combat Against Hunter Syndrome MPS II

by adminmps | Sep 6, 2022 | Clinical Trials, News, Research

We are excited to share the news that REGENXBIO Inc. has announced its intention to file a Biologics License Application (BLA) in 2024 using the FDA’s accelerated approval pathway for the treatment of Hunter Syndrome, Mucopolysaccharidosis Type II (MPS II). The...
A New Gene Therapy Trial For Hunter Syndrome (MPS II) Treatment Is Available

A New Gene Therapy Trial For Hunter Syndrome (MPS II) Treatment Is Available

by adminmps | Sep 6, 2022 | Clinical Trials, News

Homology Medicines have received clearance from Health Canada to initiate the juMPStart trial for HMI-203, a one-time, in vivo gene therapy candidate for the treatment of adults with Hunter Syndrome (MPS II). If you are interested in knowing more, join Homology...
Ultragenyx Takes Over Gene Therapy For Sanfilippo Syndrome Type A (MPS IIIA) 

MPS III Gene Therapy LYS-SAF302 Boosts or Stabilizes Development After 2 Years

by adminmps | Aug 3, 2022 | Clinical Trials, News, Research, Treatment

New data from the AAVance trial has revealed children with Sanfilippo syndrome type A, including those with severe disease, showed stable or continuously increasing cognitive, language, and motor functions two years after dosing with the investigational gene therapy...
Avrobio Receives Orphan Drug Designation for Hunter Syndrome Gene Therapy

Avrobio Receives Orphan Drug Designation for Hunter Syndrome Gene Therapy

by adminmps | Aug 3, 2022 | Clinical Trials, News, Research, Treatment

Avrobio has received orphan drug designation for its gene therapy, AVR-RD-05, from the US Food and Drug Administration (FDA) to treat mucopolysaccharidosis type II or Hunter syndrome. and is expected to initiate a Phase I/II clinical trial for MPS II in partnership...
MPS II Hunter Syndrome Latest Research:

MPS II Hunter Syndrome Latest Research:

by adminmps | Aug 3, 2022 | Clinical Trials, News, Research

Presented at the WORLDSymposium 2022 CheckRare has just posted their presentation on MPS II Research Highlights: WORLDSymposium 2022 Learn about the latest findings from recent research on Mucopolysaccharidosis type II (Hunter syndrome) with expert...
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