The September 2023 Cure GM1 Catalyst

Last Chance to Register for the Conference

There are less than two weeks left to register for the conference. Registration closes on September 11th. A Spanish translator will be available for in-person Spanish speakers. We are also excited to offer Zoom subtitle translation in 28 languages for virtual attendees.

We would love to have you join us in person or virtually to connect with others in the community and to hear talks from our amazing lineup of speakers. In-person attendees can also join a tour of Pixar Animation Studios and a baseball game between the Oakland A’s and Detroit Tigers on Saturday the 23rd. Spots are limited and are first come, first serve. See the conference agenda for details.

Move for GM1 is in Full Swing

We’re halfway through our Move for GM1 Fundraiser. Thank you to everyone participating! To show our appreciation, we’re giving away some prizes. The person who puts in the most miles in a day will win a Cure GM1 t-shirt, and the one to put in the most miles overall will win two tickets to a Disney park of their choice!

It’s not too late to join or support our movers! If you aren’t able to join as a mover yourself, you can still join the Charity Miles app to support those putting in miles. Consistency is important, so if you plan to join, make sure to post every time you move to get the best response! The more you post the higher chance you have to get donations. All donations to this fundraiser will be matched up to $10,000.

News

Cure GM1 is excited to announce a collaboration with COMBINEDBrain to create a GM1 biorepository which will help advance research and therapy development. To learn more about the partnership and biorepository, and how you can help by sending samples, please visit our biobank page.

Passage Bio Press recently announced promising interim clinical data from the first eight patients with GM1 gangliosidosis in their Imagine-1 study. Read their press release here.

Every year, the Sanofi TORCH Awards brings awareness to rare disease. The awards aim to recognize the strength and positive contributions of individuals affected by rare diseases. GM1 families have received the award 4 times in the history of the award. Some of them are featured alongside other amazing advocates in this year’s video highlighting the 2023 TORCH recipients.

Golf Fundraiser Success!

Sending a big thank you to Cure Kinley and Kennedy for hosting their third annual Golf Tournament Fundraiser. Their aim was to raise funding for critical research and development into treatments and therapies for GM1. It was a huge success, and they received over $54,000 in donations! We appreciate all the time and effort that went into putting on this big event!

Climbing Mountains for GM1

Thank you to everyone at Seven Hair & Body for making the trek to the top of Mount Snowden on August 27th. Especially appreciated are those who made the climb despite having no hiking experience! Those on the climb hiked along with and in support of fellow employee Usman, whose daughter suffers from GM1 gangliosidosis. Together, they raised over £15,000 for Cure GM1! We appreciate you! See the fundraiser here.

August was National Will Making Month

This month serves as a good reminder to always protect what you love and to ensure your family’s legacy by making sure you have a will. Freewill, in collaboration with NORD, offers a quick, easy will-making service for those connected with rare diseases. Consider including a bequest to Cure GM1 when you make yours.

Help Recognize Newborn Screening Awareness Month

Newborn Screening Awareness Month seeks to spread awareness of newborn screening, a public health service all US states provide to identify conditions that are not visible at birth, but that could lead to serious disability or death if left untreated.

Thanks to our new research collaboration with the Gelb Laboratory at University of Washington, we are working to improve the understanding of GM1 for newborn screenings. Please mail info@curegm1.org if you are in the US and willing to help provide samples from the comfort of your home.

September is Leukodystrophy Awareness Month.

Leukodystrophy awareness month takes place in September every year. GM1 gangliosidosis is a leukodystrophy / leukoencephalopathy, a group of rare, genetic disorders that affect the white matter of the brain. Help us spread awareness about these disorders for everyone with GM1.

Happy Birthday!

Eric – Aug 8
Aaron – Aug 14
Imogen – Aug 17
Jim Jim – Aug 21
Broderyk – Aug 22
Hampus – Aug 26

Ways to Give – Your Support Matters

Visit our Take Action page for more ways to support our community