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 >>
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...
The holidays are here and reading can be the ideal summer activity for children. It’s fun, portable, and can involve the whole family. Here’s a list of books for children to teach them how everyone is unique and special, how to turn feelings of sadness, anger and fear...
