Take Action
Take Action for GM1 Day
Now is the time to buy GM1 Day gear, submit for a proclamation, and get in touch with local media channels to tell your story. The more families we have talking about GM1, sharing their stories, and doing advocacy the faster we can make progress toward our goal of developing effective treatments and therapies for this devastating disease.
Contact Your Member of Congress
The FDA has created some programs to help strengthen their rare disease infrastructure, but the rare disease community does not yet reliably feel those impacts. An internal task force would be able to assess and guide the FDA’s rare disease-related processes to ensure they function optimally.
Take less than a minute to ask your Members of Congress to sign a letter to the FDA requesting they create an internal FDA task force to assess and guide agency-wide rare disease activities.
News
- The Cure GM1 2022 Impact Report is now available to read.
- Listen to Global Genes and Passage Bio discuss gene therapy and GM1 in the RARECast Podcast: Advancing a Gene Therapy for a Rare and Fatal CNS Disorder.
- Passage Bio will begin a new cohort and a new dose in the second half of 2023. Read the press release here.
- Lysogene announces the extension of deadline for the submission of offers. Read the press release here.
Community Efforts
A Cure for Violet
Violet’s family visited Sacramento, California on an advocacy visit, with the goal of having California proclaim May 23 as GM1 Gangliosidosis Awareness Day. They also got Cure GM1 included in the United for Giving program at United Health Group. Now UHG will match employee gifts to Cure GM1. Thank you for all your advocacy work!
Rare Disease Wrap-up Webinar
Thank you to Worldwide Clinical Trials for hosting the Rare Disease Day 2023 Wrapped Webinar following Rare Disease Day and including our President Christine Waggoner as a presenter. It was an honor to be included with such outstanding rare disease advocates.
Niamh’s Story
Niamh’s mother has exchanged her full-time role for a part-time one, which allows her to spend more time with and caring for Niamh as well as advocating on her behalf and on behalf of everyone in the GM1 community. She has written a touching story about their journey to inform her local community, and has also been contacting local media outlets and politicians about GM1 day. Thank you!
GM1 Day Proclamation in Idaho
Idaho has issued a proclamation for GM1 Awareness Day. Thank you Deb Ackerman for submitting! Proclamations are also in process in Virginia, Florida, Texas, Massachusetts, Wisconsin, and California. Fingers crossed they all come through! If your state is not mentioned, you can also submit for a proclamation! It takes less than 10 minutes, and we’ll provide everything you need. Email info@curegm1.org for details.
“This proclamation and others like it shine light on GM1 patients, their families, and those who have passed, including my boys, in such a way that their stories can create stepping stones of curiosity and advocacy for those who may have never been aware of this terrible disease.”
-Deb Ackerman
Face Off for Marley
A Cure for Marley is hosting Face Off For Marley Charity Hockey Game on Saturday, April 8th. If you live near Kalamazoo, you are warmly invited to attend! You can read more about Marley and the upcoming game in this Ad-visor&chronicle article. Thank you Waun family!
Coming Up
Save the Date for the 2023 International Cure GM1 Community Conference
This year, we’re hosting our first in-person conference since the pandemic. Save the date for September 22, 2023 at the Hyatt House Hotel in the San Francisco Bay Area. We would love to see you! More details to follow in the coming months.
Come to the PRONTO Natural History Study Webinar
The webinar will take place on Thursday, April 13th at 11 am – 12 pm PT. Held over Zoom, this free webinar will go over what Azafaros’ PRONTO study is, its purpose, the importance of these studies, and how those living with GM1 type 2 can participate. Speak directly to Dr. Caroline Hastings, an investigator and physician involved in GM1 research. This is an excellent learning opportunity and a wonderful way to take action and help progress the fight against GM1.
Support Cure GM1
- Make a difference and help fund scientific research with a beautiful popup card for Mother’s Day! Place your order by May 5th.
- New T-shirts and gear are on the Cure GM1 Store for Mother’s Day, Father’s Day, and GM1 Day. Buy yours now to get them in time!
- Purchase bulbs, seeds, and vegetables from Flower Power Fundraising
- Donate. Developing an effective treatment for GM1 can only happen with sustained funding. Consider pledging a monthly donation!
Happy Birthday!
- Marley – March 9
- Milan Aerts – March 17
- Armand – March 19
- Jenny – March 20
- Keeton – March 21
- Toby – March 21
- Mira – March 28
