Feb. 2017 Cure GM1 Update

This horrible disease has been in existence for far too long.  Fundraising and advocacy can change the future of this disease.  In fact, Cure GM1 has already impacted GM1 medical research.

IV Gene Therapy Update

One of the big milestones of the preclinical program is now complete.  The good news is that the meeting with the FDA was positive.  The bad news is that funding and keeping on schedule are becoming more problematic.  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.  As of now, it is becoming more likely that a clinical trial will not be initiated in 2017.   There is no one who wishes things were moving faster than the families of children who are impacted by GM1.  We must continue to push as hard as possible for continued progress and funding.  Children’s lives are at stake.  Remember, Action is HOPE!


On February 8th, 2017, Lysogene went public in Europe and received $25M in additional funding.  This funding should greatly help Lysogene and their GM1 intracranial gene therapy program.  Cure GM1 is supportive of as many safe and possibly effective clinical trials as possible, no matter what the approach.  We need as many players trying to cure GM1 as possible for the best solution to be found.  We truly wish there were even more GM1 programs in the works, but unfortunately, there are still very, very few.  The only publicly announced programs are Lysogene’s program and Cure GM1’s IV gene therapy coalition.  Continued advocacy and funding is the only way to push for the development of more research.

Let’s remember…

This is absolute worst part of the monthly update.  It has been a terrible beginning of the year.  Cooper, Ian, and Owen all passed away in just a matters of weeks.  These sweet children are now free of this devastating disease.  Cure GM1 extends our deepest condolences to all the families who have lost their sweet children.


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 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.


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.

Feb. 2017 Cure GM1 Update

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