Dec. 2016 Cure GM1 Update

Let’s bid 2016 goodbye and work towards making 2017 the year of a GM1 Gangliosidosis human clinical trial!

It’s been an amazing year for Cure GM1 in 2016!  When 2016 began we had no idea what the future would hold.  Due to the hard work of many and families joining together, we were able to exceed our 2015 fundraising efforts by raising over $600K again!  In November, we had another amazing month and over $200K was raised again!  Simply incredible.

When our daughter Iris was first diagnosed in 2013, we were told that there would likely be a clinical trial in 2016.  As you know, 2016 is almost over and there was not a human clinical trial for GM1 Gangliosidosis.  As the years have passed since Iris’ diagnosis,  we noticed a very big gap that absolutely needs to be filled.  We founded Cure GM1 to help directly accelerate a human clinical trial and to fund GM1 Gangliosidosis medical research.  Let’s work towards making 2017 the year!

We need you all!  Please consider a year end gift!

An Important First Step

This picture looks very mundane. It’s an envelope. It’s a letter. It’s more than that though. This is the very first letter to the FDA to initiate the process of truly putting the steps in motion to hold an IV gene therapy clinical trial for GM1 Gangliosidosis. We’re truly making progress. To make this a full reality, we still need help. Do you want to change history and to save children’s lives from a devastating disease? The hope is to make a clinical trial happen by the end of 2017, but we have to see what the feedback is from the FDA, etc. It’s a long time and we absolutely wish it were sooner, but for now, this is what hope looks like

Unfortunately, the manufacturing process for the therapy is quite long.  There are also a number of steps before manufacturing.  It’s important to understand that at each step, something could arise that needs to be addressed.  We certainly hope it’s smooth sailing, but the truth is that this process is complicated.  Nonetheless, we are hoping for the best and continuing to work hard towards this goal.

Fundraising

There has been tremendous fundraising and we would like to acknowledge a few folks by posting their videos.

A Cure for Clara

The Bragg family joined our nonprofit and have been working tremendously hard!  Below is a video on their story!

Jenny’s GM1 Journey

Jenny McKernan’s family  released a video and are also fundraising.  Please see Jenny’s video below.

The Leishman Family

Marilee Leishman and her husband Brad organized a benefit concert in Tucscon in honor of all the children affected by GM1 and their two sons Eli and Evan.  Check out a video from the concert below.

These are just a few highlights, but there is so much more going on.  We’re also very grateful to everyone who participated in the Cure GM1 Triple Play.  As mentioned, it was an incredible month!  We’re also extremely grateful to the team of runners who ran for Iris in the Berkeley Marathon and raised over $10K!

Just in the past few months, there have been tremendous efforts: restaurant fundraisers, cookbooks, auctions, and more!  It’s amazing!  Keep up the hard work everyone and we thank you all from the bottom of our hearts!

Please follow us on Facebook where there are many, many updates.  And please help us make this cure which is waiting in the wings, a true reality!

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Dec. 2016 Cure GM1 Update

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