June 2017 Cure GM1 Update

Fundraising Update

Almost to $2M Raised!  Less than $6000 to go!

Cure GM1 was approved in April 2015 as a nonprofit and now is extremely close to having raised $2M towards GM1 Gangliosidosis medical research.   Funds raised have supported  lentiviral gene therapy research, AAV9 IV gene therapy, and a small cyclodextrin study.  A new project is also about to be initiated for a mouse study and drug repurposing project.  More on that below in the research update.

The answers are still not fully known and as we know all too painfully, there is nothing available to patients as of now.  The mission of Cure GM1 is to help change that horrible reality.  To support research, we must continue to raise funds to advance our mission.

We are so grateful to all our supporters and generous donors!   There are many families who have joined the fight and even those who are very newly diagnosed.

We would also like to specifically acknowledge A Cure for Clara and their supporters who have been truly integral to raising funds.  GM1 families are united in this fight and our community continues to rally together.

A Cure For Clara, Thank you!

The Spring Clean fundraising campaign was a success with families who joined together and put on numerous events.  These fundraisers included races, restaurant events, a whiffle ball tournament, a school fundraiser, several yard/garage sales, a costume awareness event, and more.

Fundraising for Florence in the UK

 Little Florence’s mum Jenny Croce recently gathered a group of friends and ran a race to raise awareness and funds for Florence and to help support Cure GM1!

$2000 recently gathered from two cash boxes!

The cash donation box program also continues to help our efforts!  Please let us know if you can help by asking a business owner to add one to their store.

Research 

This month, let’s highlight a project which will involve a small company called Dorphan in Switzerland that is focused on chaperone and small molecule therapies.  Cure GM1 has agreed to help fund a mouse study to test an existing drug that was tested in humans for another disease.  This drug did not make it to market, but has established safety data in humans and may help patients with GM1.    Due to the fact that this is a repurposing project, the hope would be to accelerate the creation of a clinical trial so long as the research results in mice and other testing are successful.  See the graphic below regarding the estimated timeline.  

In Memoriam

We are very sad to report that 3 children passed away recently after courageous fights against infantile GM1 Gangliosidosis, two of whom are in direct contact with our foundation.

Bradenn, please see this link if you would like to assist the family with funeral expenses.

https://www.gofundme.com/a-final-resting-place-for-bradenn

Barron Posey, to learn more about Barron’s battle with GM1, please see the video his parents created.

We would like to extend our deepest condolences to Bradenn’s and Barron’s families.  The absolute worst way to receive donations is when our GM1 warriors pass away.  

Friends of Cure GM1

  • 2017 3rd Quarter Campaign (July – Sept.) will be a Cure GM1 Walk/Ride/Run – Stay tuned for information on this virtual walk/run coming soon during 3rd Quarter!

 

Check out the new Cure GM1 Shop!

 

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.

June 2017 Cure GM1 Update

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