The MPS group is a rare but extraordinary one with Community at our heart. 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...
Receiving a diagnosis is difficult for those who love us. In this podcast by Beyond My Battle, two caregivers provide their perspectives on what it felt like. Steve is the parent of a child with Sanfilippo syndrome and Sarah is the parent of a child with Down...
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 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...
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...
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 >>
