November News!
The Cure GM1 Catalyst
This newsletter is packed with impactful stories, helpful resources, and exciting fundraising opportunities. Don’t miss our year-end giving campaign, double your donation with our $25K match! Discover caregiver tips in our new YouTube playlist, and be inspired by families like Lucas’s and Joaquim’s, whose journeys shine a light on the resilience of the GM1 community. Explore holiday fundraising options, and stay informed on important advocacy efforts like the Creating Hope Reauthorization Act. Don’t miss our biotech update on GEMMABio’s groundbreaking partnership in Brazil. Dive in to see how you can make a difference!
ADVOCACY
COMMUNITY & ENGAGEMENT
FUNDRAISING
BIOTECH NEWS
ANGELVERSARIES & BIRTHDAYS
Cure GM1 Presents to the FDA for the Inaugural Meeting of the Rare Disease Innovation Hub
Advocacy
This meeting was organized by the FDA and the Reagan Udall Foundation at the White Oak Campus in Maryland on October 16th, 2024. The purpose of the meeting was to bring together rare disease patient advocates, academic researchers, biotech industry members, and other key stakeholders to launch the new Rare Disease Innovation Hub. Catch the full recording of the recent conference here, or jump straight to the Cure GM1 segment here to see our latest updates and insights shared with the community!
Support Needed to Pass ‘Creating Hope Reauthorization Act’ and More Information on the Rare Pediatric Disease Treatment
Advocacy
The Rare Pediatric Disease Priority Review Voucher Program has still not been renewed past December 20, 2024. Stay updated on the latest developments. Read more >
Meanwhile, H.R. 7384, the “Creating Hope Reauthorization Act” would amend the date by which the drug must be designated as a rare pediatric disease drug to September 30, 2028 and the date by which the application must be approved to September 30, 2030. This bill was introduced in the House and referred to the Energy and Commerce Committee’s Subcommittee on Health.
If the rare pediatric disease PRV program is not reauthorized, eligibility to receive a voucher will be limited to products that receive rare pediatric disease designation by December 20, 2024, and are approved by September 30, 2026.
Action Alert: Contact your Senator today to ensure the “Creating Hope Reauthorization Act” passes before December 20th, 2024!
Take Action Here>
Presentation on Patient Preference Research includes Cure GM1 at MDIC Patient Summit
Advocacy
Christine recently joined a panel at the MDIC Patient Summit to discuss patient preference research on a panel led by FDA, sharing the journey behind developing the first-ever GM1 gangliosidosis patient preference study. In collaboration with RTI International, Holly Peay, Amanda Bingaman, and our advisory board, this study still holds relevance as a groundbreaking effort to better understand GM1 patient needs and priorities.
Check out Caregiver Tips playlist
COMMUNITY & ENGAGEMENT
Check out our Caregiver Tips playlist, part of the new Caregiver Education Series on the Cure GM1 YouTube Channel! This playlist offers practical advice and insights to support GM1 caregivers, with more tips and videos coming soon. Plus, 2024 Conference presentations are now publicly available, providing valuable resources for our community. Watch, learn, and make sure to subscribe here to stay updated!
Support for Caregivers: NORD Respite Program
COMMUNITY & ENGAGEMENT
Caring for a loved one takes tremendous dedication, but a break can make a world of difference. Through NORD’s Respite Program, GM1 caregivers can access financial assistance for much-needed time away—whether to attend an event, a conference, or simply recharge for an afternoon or evening.
Read more on how to apply>
Lucas’s Story
COMMUNITY & ENGAGEMENT
Lucas’s journey with GM1 gangliosidosis has required immense resilience. He has flown over 80 hours for a gene therapy clinical trial, enduring numerous tests—from MRIs to EEGs, all with the aim of improving his quality of life and supporting future treatment options for GM1 children.
“Because of GM1, Lucas has not been able to grow up with two of his siblings”
GM1 has profoundly affected Lucas’s family. He lost two older brothers to the disease, and his sister has grown up in a household marked by caregiving and medical needs. Through these challenges, the family has found unexpected connections and compassion from their community, which have lifted them and strengthened their resolve to navigate this difficult path together.
Get to know Lucas more>
Share Your Holiday Spirit with the GM1 Community
COMMUNITY & ENGAGEMENT
As the year wraps up, we’re cooking up a festive surprise for our GM1 community, and we’d love for you to be part of it! Share a high-resolution holiday photo of your family or children, along with a meaningful holiday quote. Please note that all submissions will be shared publicly within our community. Submit by November 20th, and let’s make this season bright together! Post your holiday photo in the comments of this post or email it to info@curegm1.org. Submit your holiday quote using this form: Holiday Quote Form.
Joaquim’s Story
COMMUNITY & ENGAGEMENT
Joaquim’s journey began with hope after his family’s heartbreaking loss of his sister Sofia, who passed away from undiagnosed GM1 complications shortly after birth. When Joaquim was born in 2021, he initially showed stability, but over time his health declined. By 1.5 years old, he began experiencing respiratory issues, leading to surgeries, a tracheostomy, and eventually dependence on home care and respiratory support. Now, Joaquim faces daily challenges with frequent convulsions, requiring his parents’ full-time dedication.
In search of support and answers, Joaquim’s family chose to participate in a gene therapy study. While it hasn’t changed his condition, it has connected them with specialized medical care and offered insights that help them better navigate his needs. Joaquim’s parents remain focused on his comfort and care, strengthened by their family’s resilience and the community around them.
Get to know Joaquim & Sofia more>
We are thankful to each and everyone of you who supports CURE GM1’s work and who is part of our community.
Double Your Impact
for GM1 Families!
FUNDRAISING
From November 4th to December 3rd, your donation will go twice as far thanks to a $25K matching opportunity! This Giving Tuesday (Dec. 3rd), join us in creating hope and support for GM1 families. Your contribution will fund essential efforts in awareness, advocacy, drug development, and research, helping attract larger investments into GM1 treatment breakthroughs. Let’s make every dollar count!
Winter Flowers
FUNDRAISING
Holiday orders will not start shipping until mid November. Orders should be placed by December 1st to ship in time for the holiday
Order Here>
RaiseRight
FUNDRAISING
RaiseRight sells gift cards you can use to pay for everyday and special expenses. A percentage of the gift card goes to Cure GM1 as a donation, directly from the brand at no extra cost to you – as much as 17%! Get inspired with this interactive flipbook. From holiday prep to gifts for everyone on your list, RaiseRight is the easiest way to earn for your organization.
Read More>
Winter See’s Candies
FUNDRAISING
Treat yourself or a loved one to delicious See’s Candies chocolates this winter, and a portion of your purchase will support GM1 research. Order now and make your sweet treats count for a great cause!
Order Here>
Marley’s Miracle Mile
fundraising
Thank you to everyone who came together for the 4th Annual Marley’s Miracle Mile, a truly inspiring fundraiser created by Marley’s family to honor their journey and support! Their incredible commitment serves as a beacon of hope and shows how personal passion can drive meaningful change for GM1 families everywhere.
Inspired by Marley’s Miracle Mile? Consider hosting your own fundraiser to help us continue the fight against GM1. Every effort brings us closer to a cure and strengthens our community
Links for giving to Cure GM1:
A Cure for Marley GoFundMe
Cure GM1 Foundation
Purchase Prints and Cards to Support Cure GM1 year-round with Minted.com!
fundraising
Use the code FUNDRAISECGM1 at checkout to receive a special discount on your purchases. Plus, 15% of your purchase will be donated to charity. It’s a simple way to give back while shopping for beautiful, high-quality products. Every purchase helps make a difference for those affected by GM1 gangliosidosis.
Visit Minted.Com to support Cure GM1 >
GEMMABio Announces $100 Million Agreement with Brazil’s Leading Health Research Institute
biotech news
GEMMA Biotherapeutics, founded by gene therapy pioneer Dr. Jim Wilson, has entered a groundbreaking $100 million partnership with Brazil’s leading public health research institute, Fiocruz. This collaboration aims to advance gene therapy research and expand access to rare disease treatments in Brazil’s publicly funded health care system, Sistema Único de Saúde (SUS). Fiocruz’s investment will drive clinical research and manufacturing, making these essential therapies more accessible and affordable.Read the full story here: GemmaBio and Fiocruz Partnership.
For more details, read coverage on Endpoint News.
IN LOVING MEMORY
We extend our love and support to Jana’s family and all who were touched by her life. Jana passed away peacefully. Her strength and spirit left a lasting impact on the Cure GM1 community. We honor her memory as we continue our mission to find a cure.
