March 2017 Cure GM1 Update

While it may seem quiet at times, there is still a lot going on with our small and mighty nonprofit!  Our Cure GM1 support base is growing and we are extremely grateful.  Although we don’t wish for anyone to have to deal with GM1,  we are grateful to now have a team of families who are united in this fight.  This was by far the most active first quarter in Cure GM1 history!  There are many fundraisers occurring and it all adds up!  Every donation matters.  Even plain and simple word of mouth can make a tremendous difference.  The more people who know about GM1 Gangliosidosis, the better.  Saving children’s lives from a truly devastating disease is a truly worthy cause!  It’s only the first quarter and it’s looking to be a tremendous start!

Join us 3/25/17 at a Concert for a Cure!

Northern CA friends, please consider attending our upcoming fundraiser in Walnut Creek.  Click the link for more details.

Concert for a Cure 3/25/17 6:30pm

 

 

Cure GM1 Spring Clean 2017

We need your help!  Our small and mighty nonprofit kindly requests that you consider holding a spring fundraiser!  To read more about the Spring Clean Campaign, please click this link to read more about the campaign!  Even a simple yard sale or bake sale can make a difference!

 

 

Recent Publicity and $5000 Grant from Sanofi Genzyme

Read more about Cure GM1 in the news and about the recent $5000 grant Cure GM1 received from Sanofi Genzyme.  Also, Fundraising for Florence made a splash in the UK.  Check out these stories below!

http://www.eastbaytimes.com/2017/03/06/albany-parent-honored-for-medical-research-advocacy/

https://pjmedia.com/parenting/2017/02/28/rare-disease-day-2017-highlights-research-and-the-search-for-cures/

http://sanfrancisco.cbslocal.com/2017/02/28/bay-area-family-works-to-raise-awareness-about-rare-diseases/

http://www.manchestereveningnews.co.uk/news/florence-croce-fundraising-gm1-condition-12699610

 

 

 

 

Research Update

AAV9 IV Gene Therapy

As stated last month, it’s no longer possible that the clinical trial will be initiated in 2017.  We were operating on information provided to us early on in this process.  Unfortunately, as the story continues to unfold,  the schedule has shifted.  The new start date may be in the first half of 2018.   In every newsletter, we have written about how frustrating, complicated, and long this process is.  We are absolutely fighting for progress and we will continue to push for the process to be as quick as possible.  Forward progress continues with IND-enabling studies.    Finances and funding continue to be extremely tight.  Cure GM1 supporters’ continued generosity is required to bring this program to fruition.

Additional Research

The lentiviral gene therapy program has been initiated at UC Davis.  We expect these first 6 months to primarily be about prepping the mouse model.  There is not a lot to report on this project as of yet.

As always, we are looking into other research programs.  It’s always a good idea to consider our options and to have a multi-pronged approach.  We don’t know what the most effective solution is, so we need to keep our minds open.  While we can not disclose everything we hear, we must tell you that there is reason to hope.  GM1 research is progressing and the Cure GM1 Foundation is having an impact, even in some cases without even having the fund the projects!  There is great value in patient advocacy and creating dialogue around GM1 Gangliosidosis.

 

 

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.

March 2017 Cure GM1 Update

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