Ever wondered… How does gene therapy work?Why does it work better for some conditions that others?What is the difference between in vivo and ex vivo gene therapy?What are the ways of receiving gene therapy?How safe is it and what are the dilemmas surrounding gene...
Matthew Ellinwood, DMV, PhD, Chief Scientific Officer at the MPS Society USA, provides an overview of the mucopolysaccharidoses (MPSs) and mucolipidoses (MLs). As Dr. Ellinwood explains, most MPSs are rare genetic conditions that are often difficult to diagnose...
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...
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...
This month Rare Revolution’s online magazine recognises the unrelenting mental and physical strains of caregiving, and how essential it is that caregivers are provided with more emotional, physical and financial support to show that they are valued and appreciated. A...
