Jan. 2017 Cure GM1 Update

January tends to be a relatively quiet month for Cure GM1.  Let’s update you on what could be Cure GM1’s best year yet.   December was an excellent month in terms of donations, and November and October were off the charts.  We are still waiting for the corporate matches from the 4th quarter to arrive.  The corporate matches from the fourth quarter will significantly advance our progress towards our goal.  In 2016, Cure GM1 raised over $700,000 which exceeds our 2015 fundraising by over $100,000.

Let’s set our hopes high and shoot for the stars for 2017.  Let’s make this trial happen by the end of 2017!  This horrible disease has been in existence for far too long.  Fundraising and advocacy can change the future of this disease.

IV Gene Therapy Update

We are still hopeful that the end of 2017 might still be the year of an IV gene therapy clinical trial for GM1.  There is an upcoming meeting with the FDA and the FDA feedback will be absolutely critical in terms of determining the course of the program.   Fingers and toes crossed for positive feedback!  For now, there is really not very much to report otherwise.  Honestly, this process is painfully slow and dealing with the FDA is just one facet of this process.   Let’s continue our hard work and let’s work towards saving children’s lives NOW!

 

Let’s remember…

It’s so very sad to realize that children are continuing to lose their lives to this horrible disease.  In particular, the Type 1/infantile GM1 Gangliosidosis is so very severe.  Our hearts go out to all the sweet angels whose battles with GM1 ended in 2016.  Please honor the memories of the sweet angels listed.  Sadly, there are likely many more children who passed away, but are not in contact with our foundation.

Madilyn Rose Perry 8/30/13 – 2/19/2016
Rayan Louchahi 1/13/2015 – 4/23/2016
Paola 5/2016
Jaxson Reish 7/28/2014 – 6/16/2016
Maykel Cal Oliveira 7/11/2016
Lucy Day 7/22/2016
Maite Ugarte 5/1/2014 – 11/2/2016
Porter Heatherly 9/14/2012 – 11/10/2016
Kierra 9/1/2015 – 12/28/2016

Rare Disease Day, February 28th, 2017

Want to help raise awareness for rare diseases and GM1 Gangliosidosis?  Rare Disease Day is February 28th 2017 and it is the perfect time to raise awareness, to fundraise and to contact the press to bring more attention to our cause.  Wear denim and an awareness t-shirt!  Ask friends to do so as well!

 

 

Cure GM1 Donation Box Project

This fall, we initiated a program for donation boxes at local businesses.   So far, so good and we would love to expand the program.  Do you know a business owner who might be willing to help by adding a donation box in their store?  Over time, these small donations can really add up.  Cure GM1 can help customize the imagery for the box to personalize the stories for particular children and families as well.  Please contact us if you would be willing to help with this effort!  We currently have 27 boxes out there!  One family has raised several thousand dollars from just 2 boxes.  Imagine what we can do if we can distribute even more!  Please contact us for more information if you can help.

 

 

Cure GM1 Registry

To attract bio tech companies, we need to find as many people who suffer from GM1 Gangliosidosis as possible. Furthermore, the more concrete data, the better.  Information about the patient population can impact trial design and the level of interest from bio techs.  Rest assured, GM1 is a rare disease, but we’re already in contact with far more families than expected.  Let’s keep improving the registry and get in touch with more GM1 families!  All personal identifying information is anonymous unless specific permission has been granted otherwise.

 

Every Donation Matters

Your donation today will help save children’s lives.

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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.

Jan. 2017 Cure GM1 Update

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