Bone involvement is one of the most prevalent aspects of GD and a major cause of pain, disability, and reduced quality of life. All three types of Gaucher disease have bone disease in common and can have serious complications in those …
Sharing our latest newsletter which includes details of our first webinar for 2021, a message of thanks from a young Jordanian patient and the publication of our latest GD booklet to mark Rare Disease Day 2021
You can read the newsletter in full …
To mark Rare Disease Day 2021, we are pleased to publish our new information booklet on Gaucher disease Types 1, 2 and 3: DOWNLOAD THE BOOKLET HERE
We have developed this booklet as a source of information for patients and their carers and to …
As many countries are still in lockdown, people including those living with Gaucher disease are spending a lot of time at home. The IGA is supporting its members in these difficult times with a webinar: ‘Wellness for …
PR001 in development by Prevail Therapeutics
Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company, is developing an investigational gene therapy called PR001 that has the potential to slow or stop the underlying …
Our first newsletter of 2021 is now available to read, sharing with you the opportunity to get involved in our Gene Therapy focus group and a joint statement with the EWGGD on COVID-19 vaccinations for patients with Gaucher disease.
The IGA is looking for Gaucher patients and parent/caregivers to join a focus group to discuss gene therapy. We are seeking to understand the concerns and questions the community has around this potential new treatment. The focus group will last for …
The EWGGD and IGA are standing together with the Gaucher patients’ community in this challenging time of the COVID-19 pandemic, and we are strongly supportive of the vaccination program for COVID-19 and hopes that any patient who is offered …
A paper on a Charitable Access Program for patients with lysosomal storage disorders in underserved communities worldwide has now been published online on the Orphanet Journal of Rare Diseases (OJRD) website.
IGA's CEO, Tanya Collin-Histed …