Get Ready for an Action-Packed Month with Cure GM1
The Cure GM1 Catalyst
This month is filled with exciting events and opportunities for you to engage with the Cure GM1 community! From our highly anticipated Annual Virtual Community Conference to crucial advocacy efforts with the FDA, there’s something for everyone who’s passionate about making a difference in the fight against GM1 gangliosidosis. Our conference will feature expert speakers and an agenda packed with valuable insights, while upcoming fundraisers and initiatives offer meaningful ways to contribute to our mission. Read on to discover how you can get involved and help us drive progress toward a cure!
Community Engagement
Fundraising
Advocacy Events and Opportunities
Biotech News
Angelversary | Birthday
Join Us for the Cure GM1 Foundation’s Annual Virtual Community Conference This Month!
COMMUNITY & ENGAGEMENT
On September 20th is your chance to connect with a global community dedicated to the fight against GM1 gangliosidosis. The Cure GM1 Foundation’s Annual Virtual Community Conference brings together families, researchers, and biotech companies, creating a powerful platform for advocacy, support, and the advancement of drug development. We invite families to attend the conference at no cost. Don’t miss out on this unique opportunity to be a part of something truly special. Register today and join us in the fight against GM1 gangliosidosis!
Move for GM1 Raises $25K+
A Huge Thank You to Our Community!
We are excited to announce the incredible success of the Move for GM1 fundraiser, where our dedicated families, supporters, and friends came together to raise an impressive $25k in support of GM1 gangliosidosis research and awareness. Your participation, whether through walking, running, cycling, or sharing our mission, has made a significant impact in the fight against GM1. Special thanks to all participants!
We are inspired by the outpouring of support, and we want to express our heartfelt thanks to everyone who contributed. There were many highlights and we are grateful to all.
1. Thank you to the anonymous $5,000 gift made in honor of Clara, a brave GM1 warrior.
2. Niclas Flysjö ran at least 5 miles every single day and completed his campaign with his second ever marathon. His first marathon was last year for Move for GM1, honoring his three children and all those who suffer from GM1.
3. Aidan’s mom added her own Twitch streaming fundraiser and Aidan’s grandmother also joined in the Move for GM1 campaign, together raising $3975!
Sanofi TORCH Award Honors David Law in Memory of Violet and the Global GM1 Community
COMMUNITY & ENGAGEMENT
The Cure GM1 Foundation is honored to share that David Law, a dedicated advocate in the GM1 community, has been awarded the 2024 Sanofi TORCH Award in memory of his daughter, Violet.
This recognition is a poignant tribute to Violet’s courageous spirit and the tireless efforts of her family to advance the fight against GM1 gangliosidosis. Although Violet’s passing remains deeply felt, her legacy lives on through the advocacy, fundraising, and support that her life inspired.
The Law family has been unwavering in their commitment to the rare disease community, channeling their grief into a powerful force for change. Their work with the Cure GM1 Foundation continues to drive awareness and accelerate the search for treatments for GM1 gangliosidosis.We extend our heartfelt thanks to Sanofi for this significant honor and to everyone who has supported the Law family and the Cure GM1 Foundation in our shared mission. Together, we continue to strive for a future where no family has to endure the pain of losing a child to GM1. Watch Full Video>
The Inn Provides Calm While Study Provides Time
COMMUNITY & ENGAGEMENT
A Cure GM1 family with three siblings—Hampus, Isabella, and Julia—who all suffer from a rare genetic disorder received intravenous AAV9 gene therapy as part of their treatment. This groundbreaking therapy aims to address the root cause of their condition by delivering a corrected version of the faulty gene. For more about their journey and how they are coping with this rare disease. Read full story>
Aidan’s Story and Outstanding Advocacy
COMMUNITY & ENGAGEMENT
Aidan’s journey with GM1 gangliosidosis is not just a story of resilience but also one of advocacy and community involvement. Recently, Aidan and his family took part in Rare Across America, a program that connects individuals affected by rare diseases with their legislators to advocate for critical policies and support.
Aidan was diagnosed with GM1 gangliosidosis at a young age, and his family was suddenly thrust into the challenging world of rare diseases. Despite the many obstacles they faced, Aidan’s resilience and spirit have been a source of strength for everyone who knows his story. His family has been tireless in their efforts to raise awareness about GM1 and to support the mission of the Cure GM1 Foundation.
Their advocacy work has not only brought attention to GM1 gangliosidosis but has also united a global community of families, researchers, and supporters. Through their fundraising efforts, Aidan’s family has helped fuel the research that is crucial for finding effective treatments and, one day, a cure.
As we share Aidan’s story, we are reminded of the importance of every voice in this battle. Each child’s journey adds urgency to our mission and deepens our commitment to the work that lies ahead. Aidan’s courage inspires us to continue fighting for a world where no family has to face the challenges of GM1 gangliosidosis alone. Read Aidan’s Story Here >
Urgent: Rare Pediatric Disease PRV Program Renewal Expiration on 9/30!
Advocacy Events and Opportunities
The Rare Pediatric Disease Priority Review Voucher (PRV) program is a critical initiative that incentivizes the development of treatments for rare pediatric diseases. However, the current renewal for this program is set to expire on September 30th, 2024.
This program has been a lifeline for many in the rare disease community, including those affected by GM1 gangliosidosis, by encouraging biopharmaceutical companies to invest in developing new therapies. The expiration of this program could significantly impact the momentum we’ve gained in rare disease research and development.
We urge everyone in our community to stay informed and take action. For more detailed information about the PRV program and its impending expiration, visit Rare Disease Website>
Together, we can continue to push for the renewal of this vital program and ensure that life-saving treatments for rare pediatric diseases remain a priority. Read More>
Advocate for GM1: Join the Crucial FDA Meeting
on October 16th!
Advocacy Events and Opportunities
Join us on October 16th for an important public FDA meeting hosted by the Rare Disease Innovation Hub. This event is a crucial opportunity to engage with the FDA on advancing therapies for rare diseases like GM1 gangliosidosis. Your participation is vital in amplifying our community’s voice. Mark your calendars and join us in advocating for the GM1 community!
Event Details:
Date: October 16, 2024
Event Link: Advancing Rare Disease Therapies Through the FDA Rare Disease Innovation Hub
Cure Kinley and Kennedy’s Golf Fundraiser: $57,561.81 Raised – An Unforgettable Day!
FUNDRAISINGOn Sunday, August 4th, Cure GM1 had the pleasure of witnessing an extraordinary outpouring of support at the 2024 Cure Kinley & Kennedy Golf Tournament. Over 200 participants joined forces with us, and we’re excited to announce that the event surpassed our fundraising goal, raising an impressive $57,561.81!
We at Cure GM1 are deeply grateful to everyone who participated, donated, and made this event a resounding success. Your dedication and generosity mean the world to Kinley, Kennedy, and the entire GM1 community.
Last Chance for Fall Bulb Orders!
FUNDRAISING
Don’t miss out on your opportunity to order fall bulbs and support a great cause. Place your order now before it’s too late. Order Here>
Plan Your Year-End Giving!
FUNDRAISING
As the year comes to a close, it’s the perfect time to start planning your charitable contributions. Mark your calendars—Giving Tuesday will be on December 3rd in 2024. Let’s make a difference together!
Fall See’s Candies
FUNDRAISING
Treat yourself or a loved one to delicious See’s Candies chocolates this fall, and a portion of your purchase will support GM1 research. Order now and make your sweet treats count for a great cause!
Order Here>
Support Research on Newborn Screening for GM1
COMMUNITY & ENGAGEMENT
If your child was diagnosed with GM1 gangliosidosis as an infant, participating in this study could be a meaningful way to help researchers improve newborn screening practices. The Parent Voices on Newborn Screening initiative is seeking parents or caregivers of children who have received a positive or uncertain newborn screening result since May 2021. Your story could contribute to better communication of screening results to families. Participants will be involved in a 1-2 hour interview and a brief questionnaire, with a $50 gift card offered as a token of appreciation. To learn more and see if you’re eligible. Download PDF Here>
GEMMABio Launches New Website
biotech news
GEMMABio is excited to announce the launch of their new website, offering a comprehensive look at their innovative work in gene therapy and biotechnology. The site provides detailed information on their cutting-edge research, the latest advancements in their therapeutic pipeline, and insights into their mission to transform lives through science. Explore the new website at GEMMABio to learn more.
