Rapsodi Patient Engagement Meeting May 2019
For those of you who were unable to attend the recent Rapsodi Patient Engagement Meeting held in London; we are very please to share their speaker videos from this event, including our very own James.
If you are a Gaucher patient or family member of a Gaucher patient who has not already signed up for this study but after watching the videos would like to get involved,full details are below.
Rapsodi newsletter May 2018
Have you enrolled yet onto the RAPSODI online study?
Ambroxol clinical trial begins at the Royal Free and University College London.
The research team at the Royal Free Hospital and University College London,led by Professors Anthony Schapira, Atul Mehta and Derralynn Hughes have over the last 6 years been investigating variation in the GBA gene and what it can tell us about Parkinson’s. People with Gaucher and some of their family members carry the GBA gene and there is a slighter higher chance of developing Parkinson’s in later life if you carry this genetic alteration, but this is still a very low risk and most people do not develop Parkinson’s. The research team is currently observing associated patterns of movement, memory, sleep, mood and sense of smell using an online study portal. The aim is to intervene much earlier before the movement symptoms associated with Parkinson’s ever develop and to protect the nerve cells in the brain.
With the generosity of time and help of people with Gaucher their research has now led to a phase II drug trial of Ambroxol, a drug traditionally used as a respiratory medication which has been showing very promising neuroprotective qualities.
Recruitment started last year in 2016 on the RAPSODI study.
So far over 200 people with Gaucher disease and their families have done the online portal, mainly from the Lysosomal Storage Disorders Unit at the Royal Free in London. You can visit the study portal to see more about the research www.rapsodi.org.uk People are now being invited to take part from the other centres around the country and the team will be visiting Cambridge, Salford and Birmingham in the next few months. The study takes about 45 minutes to do on your computer or laptop at home and then some postal tests.
HOW TO GET INVOLVED
If you would like to take part or learn more please contact Dr Marco Toffoli firstname.lastname@example.org 02080168177 or visit https://rapsodistudy.com/en