Over $750K+ Raised in the first 2 quarters of 2017!
Summer is here and June was another AMAZING month! The next few months (July and August) will likely be relatively quiet months for fundraising, but the 4th quarter is typically quite robust. Still, please feel free to have a lemonade stand or do a summer event to help Cure GM1!
More information coming soon regarding our Walk for a Cure event and other events!
Cure GM1 is making meaningful progress. In late 2013 when our daughter Iris was first diagnosed, there was really hardly anything to discuss in terms of the prospects of cures. We scoured the internet and there were just a few nascent projects. The prospects of a cure are truly more hopeful now. There are several projects where clinical trials seem to be nearing.
The research continues to progress on multiple fronts, but as you may have observed, it feels very slow for dying children who are in desperate need. Nonetheless, we are still fighting and we are still hopeful.
The Dorphan project is currently looking to be the quickest path forward since the project involves drug re-purposing and the human safety data for the drug already exists.
Lysogene has also made several big steps recently and advanced their GM1 gene therapy program in several respects. Lysogene now has a manufacturing contract to produce the vector for the trial. Furthermore, they have completed the FDA and EMA orphan drug designation process for their AAVrh10 gene therapy. This is also positive news and has been building since their collaboration with Auburn and UMass was first announced in 2015. The Lysogene web site currently says clinical trials are expected in 2019.
To learn more:
A Cure for Clara in University of Texas Alumni Magazine Reaching Over 100,000 Readers
The Bragg family has now raised over $1M towards GM1 Research! Simply amazing!
Read the story here: https://alcalde.texasexes.org/2017/07/a-cure-for-clara/
Similar Rare and Amazing Stories
The stories of those who suffer from rare diseases are extremely powerful and meaningful. Below are some stories about other children with other rare diseases. Read more to be inspired and to brainstorm ideas on how we can further the mission to Cure GM1!
Save Purnell – Niemann Pick A, lysosomal storage disease very similar to GM1
Charlie Gard, International Advocacy for Rights to Seek Treatment
When you fight hard enough and loudly enough, the world can take notice! While this story is extremely emotional and just heartbreaking, Charlie’s story is that of every child suffering from a rare disease. Desperate parents are fighting for treatment for their children. Children with GM1 Gangliosidosis and all those suffering from rare diseases deserve a chance at life.
Every Donation Matters
Your donation today will help save children’s lives.
All donations are tax exempt and we will send you a receipt for your tax records so long as the necessary contact information is provided. There are no paid employees at Cure GM1 and the maximum amount humanly possible is being put towards saving children’s lives.