Rare Disease Day 2018
Congratulations to Cure GM1 board member Ryan Bragg and his wife Jenny on winning the Sanofi Genzyme TORCH award for outstanding patient advocacy to advance awareness and treatments for lysosomal storage diseases. To read more about A Cure for Clara and the Bragg family’s heroic efforts, please see the announcement published by Sanofi Genzyme.
Sweet Iris‘ 10th Birthday, Rare Carousel of Possible Dreams and Rare Disease Day events!
Cure GM1 was founded by Iris’ family and Iris turned ten years old on February 19th. In honor of her birthday and Rare Disease Day, her family participated in the Rare Carousel of Possible Dreams event hosted by Global Genes and the Festival of Children and 30+ other rare disease nonprofits. Fundraising throughout February, on Iris’ behalf raised $38K for GM1 research and included KRON4 TV coverage on Rare Disease Day and an op-ed.
Join the Fight!
A special thanks to little Lorena’s family/the Mendes family who honors Lorena’s memory through advocacy in collaboration with PBR. Those impacted by GM1 all have the power to advocate for treatments, funding, and change. Please join the fight and help us defeat this awful disease.
The WORLD Symposium for lysosomal storage diseases included several posters specifically on GM1 Gangliosidosis. Below are pictures of the posters and links to the abstracts of the associated publications.
Distinct progression patterns of brain disease in infantile and juvenile gangliosidoses: Volumetric quantitative MRI study
More advocacy and more research into GM1 is needed. None of the presentations at the conference involved clinical trials for GM1 or treatment techniques applied to GM1. The only way to push towards treatment is through more advocacy and funding.
We hope to announce our next series of grants and new projects in the coming months. To continue to advance HOPE, we require your assistance.
In Loving Memory
Two sweet children passed away on the very same day on February 8th, Hanna from Romania and AJ from New Zealand.