Video: Mucopolysaccharidoses and Mucolipidoses Explained
Dr. Matthew Ellinwood provides an overview of the mucopolysaccharidoses (MPSs) and mucolipidoses (MLs).
A New Gene Therapy Trial For Hunter Syndrome (MPS II) Treatment Is Available
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...
Canadian MPS Society AGM
The 2022 Canadian MPS Society Annual General Meeting will be held on: Monday 26 September 2022
We need YOUR VOICE!
Join our incredible team of Directors in 2022 to help oversee the organisation’s strategic purpose and direction. Your leadership can have a life changing impact on the lives of families affected by MPS.
Podcast: How To Manage the Responsibilities of Being a Caregiver and Avoid Burnout
In this podcast by Beyond My Battle, learn how any caregiver can address feelings of helplessness, how to support a loved one effectively, and the importance of practicing self-care to avoid burnout.
MPS III Gene Therapy LYS-SAF302 Boosts or Stabilizes Development After 2 Years
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 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:
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...
Current Treatment Options for MPS I and Gene Therapy
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 >>
A Guided Pathway to the Clinical Trials Process
Courageous Parents Network has a Guided Pathway designed to present families with a clear-eyed picture of the clinical trials process, through the lens of families, researchers and professionals, and to empower you with information and perspective to help you...