Goodbye July & Hello August.
The Cure GM1 Catalyst
Welcome to this month’s newsletter! We’re excited to introduce Ashlei Brittany as our new Conference Coordinator and Tayara as Program and Development Manager, bringing fresh perspectives to our mission. Two important milestones recently occurred. The NIH team published ten years of natural history and Gemma Biotherapeutics has acquired the rights to carry forth the UPenn / Passage Bio GM1 gene therapy program. Read more below. As we prepare for our virtual conference, we invite you to engage by sharing photos and quotes for our conference video. As always, we invite you to participate in helping to create meaningful change. Please join us in August for our Move for GM1 campaign and $10K matching offer and there are always volunteer opportunities. Stay connected for more updates and support as we navigate the challenges and milestones ahead, united in our fight against GM1 gangliosidosis.
Community Engagement
Advocacy Events and Opportunities
Biotech News
Fundraising
Angelversary | Birthdays
Move for GM1: 10K Fundraiser Match
FUNDRAISING
Move for GM1 is a simple and impactful fundraiser taking place this August, and there’s still time to join! Participants log their miles—whether walking, running, biking, or any other activity—using the Charity Miles app and share their progress on social media to encourage donations. With a $10K match on the line, every contribution counts.
Need help or have questions? Every little bit helps and matters in advocating for those with GM1 and raising money for Cure GM1. Join us today and make a difference!
To Participate in Move for GM1 download the Charity Miles App
To participate, download the Charity Miles app, click the “Get Sponsored” button, and add a photo. Throughout August, log your miles in the app and share your progress on social channels to inspire donations. You can accept contributions via the Charity Miles app or our GiveButter Team Campaign, both of which accept donations from most countries.
Download Charity Miles >
exciting team updates
COMMUNITY & ENGAGEMENT
We’re thrilled to announce some exciting team updates! Please join us in welcoming Ashlei as our new Conference Coordinator and Tayara as Program and Development Manager, and celebrate Anita’s transition to the Communications role. These changes promise to inject new energy and expertise into our mission at Cure GM1!
Ashlei Brittany
Community Conference Coordinator
Ashlei Brittany joins the Cure GM1 Foundation as the (Contract) Community Conference Coordinator. With a bachelors in Entertainment Business, Ashlei has been planning events in the entertainment and nonprofit industries for her entire career. As a rare disease patient, she has devoted her career to helping others and finding any way to make the world a better place. In recent years Ashlei has held roles in fundraising, engagement, and client services, utilizing her expertise to help those around her. She is excited to bring her event planning and communication skills to Cure GM1 to help with the annual community conference this September.
Tayara Calina
Program and Development Manager
Tayara joins Cure GM1 Foundation as Program and Development Manager, bringing over 13 years of experience in social impact project management and public policy. Her career is built on driving meaningful change through collaboration, relationship building, and strategic design. In her most recent role, Tayara served as a public policy coordinator for a tech company in Brazil, where she led state and local political engagement, advocating for public safety and driver welfare while building public-private partnerships. Previously, at a philanthropic institute, she led capacity-building projects for civil society and established a network of cities dedicated to public sector innovation and economic development.
Facing GM1: A Family’s Journey Towards Hope
COMMUNITY & ENGAGEMENT
In the heart of a family touched by GM1 gangliosidosis, the initial shock of diagnosis gave way to a sense of direction. “We were devastated, with my wife in tears for months,” shares Lingxi’s father. “But understanding what we were dealing with has helped us focus on how best to support our child.” Their journey began with countless therapies, from dance lessons to intensive physical sessions, aimed at overcoming their child’s developmental delays. Despite their tireless efforts and financial investment, progress remained elusive, highlighting the relentless nature of GM1.
GM1 gangliosidosis has significantly impacted their child’s life, leaving her struggling with motor skills, speech clarity, and concentration—challenges that intensify as she grows. “Our lives are a continuous adaptation to her needs, always hoping for stability yet often facing regression,” Lingxi’s father explains. This personal story underscores the daily realities for families grappling with rare diseases: a blend of hope, adaptation, and the constant pursuit of normalcy amidst profound challenges.
The family’s narrative is a poignant reminder of the critical role of Cure GM1 Foundation. By bridging the gap between patients and researchers, the foundation fosters a community of support and information, offering a beacon of hope for those navigating similar paths. “Supporting the Cure GM1 Foundation is essential—not just for our family but for all humanity,” asserts Lingxi’s father. “Together, we can advance research and possibly conquer this and other rare diseases, ensuring future generations can thrive without the shadows of such conditions.”
Read Lingxi’s Story Here >
Dr. Cynthia Tift publishes ten years of GM1 natural history data
COMMUNITY & ENGAGEMENT
We congratulate Dr. Cynthia Tifft and her team at the National Institutes of Health for their significant achievement in publishing ten years of GM1 natural history data. This important research provides invaluable insights into the progression and impact of GM1 gangliosidosis over a decade, offering new understandings that could pave the way for future treatments.“Thank you to all the families who made this work possible!” -NIH Team To explore their findings and the implications for those affected by GM1.
Read Results of 10-year GM1 study >
VOLUNTEER OPPORTUNITY
COMMUNITY & ENGAGEMENT
Lend your expertise to Cure GM1! We’re seeking volunteers skilled in website support, marketing, blogging, graphic design, public relations, and video editing. Your contribution can make a significant impact in our fight against GM1. Join us in our mission to make a difference!
Contact us at info@curegm1.org.
Participate in the COMBINEDBrain Biorepository Collection Event on September 29th
COMMUNITY & ENGAGEMENT
Join us for a pivotal Biorepository Collection Opportunity on September 29th, 2024, at the Kansas City Marriott Country Club Plaza. Organized by COMBINEDBrain, this event invites patients to contribute samples that will support vital research. If you or someone you know is interested, please register your details through our Conference Participation Form. and be a part of this crucial initiative to advance scientific understanding.
The COMBINEDBrian Conference Participation Form Registration >
Navigating Back-to-School Grief: Support for Families Facing Loss
COMMUNITY & ENGAGEMENT
As families prepare for the back-to-school season, those grieving the loss of loved ones face unique challenges. The onset of a new academic year often brings poignant reminders of milestones that won’t be reached and the passage of time without their loved ones. For insight and support during this emotionally charged period, visit Grief Beyond Belief for resources and guidance on navigating grief during back-to-school transitions.
Grief Beyond Belief >
FDA Rare Disease Innovation Hub Created
biotech news
“An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children. Many rare conditions are life threatening, and most do not have approved treatments. Fundamental to the mission of the U.S. Food and Drug Administration is to engage patients and caregivers – to understand their unique perspectives and experiences and keep these front of mind as we review medical products for rare disease patients.”
Read More about Rare Disease Innovation Hub >
Azafaros Press Release
biotech news
“Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C. The trial was conducted across three sites in Brazil, involving 13 patients older than 12 years of age, with the aim of determining the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat. The results will guide the identification of the target dose for Azafaros’ planned Phase 3 pivotal studies.
Read more >
GEMMA Biotherapeutics Press Releases and Departure of Dr. Jim Wilson from UPenn
biotech news
Granted exclusive, worldwide rights for the development and commercialization of PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease, and PBML04 for metachromatic leukodystrophy to GEMMA Biotherapeutics, a new company co-founded by Dr. James M. Wilson. Entered new strategic research and collaboration agreement with GEMMA Biotherapeutics to advance genetic medicines for CNS indications, including Huntington’s disease.”
Read more >
Attend this Year’s 2024 International GM1 Virtual Community Conference
advocacy events & opportunities
Secure Your Spot: Register by August 15th for a Rare Bear Surprise
Register for the Annual Conference by August 15th and receive a special Rare Bear in your conference box! These unique bears are a symbol of support and unity within the rare disease community.
Secure your spot at Rare Science >
Share Your Story: Contribute to Our Tribute Video
Help us craft a moving tribute for our conference opening video by sharing a quote and photo of your loved one. Whether celebrating their life or honoring their memory, your contribution will touch many. The video will be featured at our conference and across our social platforms. Participate in this heartfelt project by emailing info@curegm1.org or submitting your story. Submit your story >
RARE Advocacy Summit
advocacy events & opportunities
Join the rare disease community in Kansas City, Missouri, for the 2024 Week in RARE, hosted by Global Genes. This pivotal event integrates the RARE Health Equity Forum, RARE Advocacy Summit, and the RARE Champions of Hope awards, alongside annual membership meetings for the Global Advocacy Alliance and RARE Corporate Alliance. Don’t miss this unique opportunity to connect, engage, and share experiences with fellow advocates and leaders dedicated to making a difference in the rare disease sector. More info on RARE Advocacy Summit >
The NORD Rare Diseases and Orphan Products Breakthrough Summit
advocacy events & opportunities
The NORD Rare Diseases and Orphan Products Breakthrough Summit is an essential gathering for those connected to the rare disease community, bringing together leaders from patient advocacy groups, industry, academia, and government. The 2024 Summit, themed “Equitable Access to Innovation,” will delve into the latest in clinical trials, gene therapy, medical devices, AI, and patient-focused drug development, emphasizing inclusivity and equal access. This event offers unparalleled networking opportunities and a chance to explore cutting-edge research in the Poster Hall.
Learn more about NORD and get involved >
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 >
Beautify your Garden and Contribute to a meaningful cause
fundraising
Now through October 15th, support GM1 research, advocacy, and drug development by purchasing bulbs and flowers at . With 40% of proceeds donated, your purchase helps fund vital efforts to find a cure. Beautify your garden while contributing to a meaningful cause. Shop today and make a difference!
Purchase Bulbs & Flowers today >
