Inform Rare has launched a new resource – CommuniKIDS – a template to help researchers convey clinical trial results back to participating youth and their families in plain easy to understand language. CommuniKIDS, funded in 2021 by CHILD-BRIGHT, takes its...
Grief is considered a “taboo” topic and should be avoided in conversation. As a result, even fewer people know how to respond to and process grief – both their own and that of others. Grief can be experienced not only after someone’s passing but also can...
Listen to a talk about the sibling perspective and the support available with Emily Holl, the Director of the Sibling Support Project. Emily is a social worker, writer, trainer, and sibling. Over the past 16 years, she has provided workshops, training, and groups for...
Want to find out more about anaesthesia and how it relates to rare diseases? OrphanAnesthesia publishes anaesthesia recommendations for rare diseases in order to facilitate the work of anaesthesiologists and to improve patients’ safety. This a project of the...
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...
