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

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...
Current Treatment Options for MPS I and Gene Therapy 

Current Treatment Options for MPS I and Gene Therapy 

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

In this radio interview, Dr. Wang discusses the current treatment options for MPS I as well as the work he is doing to assess the safety and efficacy of gene therapy (RGX-111) for this rare disease.  Listen to the interview >>
NICE Recommends Vimizim For Treating MPS IVA Morquio

NICE Recommends Vimizim For Treating MPS IVA Morquio

by adminmps | Apr 29, 2022 | Clinical Trials, News, Research, Treatment

We are excited to hear that Elosulfase alfa – branded as Vimizin and made by BioMarin – has been recommended by NICE (National Institute for Health and Care Excellence) for treating MPS IVA Morquio in England and Wales. Newly diagnosed patients and...

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