Rare Disease Day

is on February 28, 2023. Leading up to the day, there are lots of ways to take action, including participating in a photo contest for a $50 gift card, sharing GM1-related stories and information on social media. Update your profile frame now with our 2023 design. On February 28th, we encourage you to wear stripes, blue jeans or some of our awesome Rare Disease Day gear and post on social media. Encourage your friends and family to do the same!

Share Your Story

A notable and worthwhile way to take action for Rare Disease Day is to share your personal story with your community. Contact your local newspapers, magazines, and TV stations to see if they’d be interested in covering you and your family in a human interest story. Feel free to use our media contact letter template when you reach out!

Rare Disease Portraits in Times Square

If you are in New York, Beyond the Diagnosis will feature portraits of children with rare diseases in Times Square including, Sweet Iris. The Jumbotron is a double-sided screen 5 stories above the northeast corner of the intersection at 1500 Broadway & West 43rd Street. Send us photos of the Jumbotron if you see it!

GM1 Day is in May

While GM1 day is still months away, now is the time to start mailing your state and governor about recognition for GM1 Day. If you’re interested we can help you look up your state information and provide material with which to apply. Please email info@curegm1.org for more information.  

Support Cure GM1

  • Use the Round Up App when shopping to round up your purchase to the next dollar and give the difference to Cure GM1.
  • Walk or run with Charity Miles. Download the app and choose Cure GM1 as your charity of choice. Be sure to ask friends and family to donate to your campaign.
  • Take advantage of the last weeks to shop with Amazon Smile to support Cure GM1. The service will no longer be available starting February 20, 2023.
  • The Walmart RoundUp program is also available to use while shopping on Walmart.com.

Valentine’s Gifts

Our simple gifts are the perfect way to remind your loved ones they are close to your heart – purchasing them also supports Cure GM1’s mission to develop effective treatments and therapies for GM1.

  • TEGA Therapeutics will present a poster on progress in the development of enzyme replacement therapy for GM1 funded by Cure GM1 at the WORLDSymposium 2023. 
  • Passage Bio indicated in a recent article and interview with the CEO that the dose for their study will be increased and that clinical trial criteria will be adjusted. The expansion study has not yet begun and no new patients are being enrolled.
  • Now is the time to join a natural history study! Both the UPenn study and the Azafaros study are currently enrolling.
  • Families are encouraged to register for FREE for the WORLD Symposia Meeting. Cure GM1 will be attending and catching up with biotechs and researchers from around the globe.  Better yet, Christine Waggoner, our President will be honored as the 2023 recipient of the Patient Advocacy Leadership Award.
  • In January, members of the Cure GM1 board attended the Rare Affair event at the J.P. Morgan Healthcare conference. It was a great opportunity to connect with biotech companies working in rare diseases and to network!
  • Behind the scenes, Cure GM1 has been hard at work navigating critical issues with respect to GM1 natural history data sharing and assessing new programs to further patient advocacy efforts. All your support helps us fund this critical work which could help biotechs and all GM1 programs.

February Caregiver Support Group

Dr. Al will be back on February 21st at 3pm PT  to lead another virtual caregiver support group. Please come to this comfortable space where you can listen and share with other GM1 caregivers who understand your experiences. Email info@curegm1.org to be added to the invitation.

RARE Compassion Program

Apply to participate in the RARE Compassion program. In this program, your family will pair with a medical student, whose goal is to learn about your unique needs and challenges as well as build compassion and understanding for those living with rare disease. For you, the program is an opportunity to advocate and spread awareness for the GM1 community, as well as gain a greater understanding of the medical system.

Orphan Drug Act

According to the Every Life Foundation, the Orphan Drug Act Celebrated its 40th anniversary on January 4th. According to the Every Life Foundation, “the ODA has resulted in the development and approval of approximately 700 new drugs and biologics for rare diseases. These therapies have delivered life-saving and life-changing outcomes to thousands of children and adults.”

Sing Me a Story

At the Sing Me a Story Foundation “volunteer musicians create music inspired by the creative stories of children. These songs and stories raise awareness and financial aid for nonprofits and provide families with an unforgettable experience that brings joy and healing.”

Contact Sing Me a Story to request a story for your child! 

GM1 Information Sheet

Take a look at our new GM1 gangliosidosis information sheet, a helpful resource for those new to GM1. Bookmark it and share with anyone who wants to learn more about the disease.

Happy Birthday

  • Vittoria Anne – Jan 02
  • Lucas – Jan 09
  • Adeena – Jan 30

A special Happy Birthday to Florence who turned nine on January 30th. And thank you to Andrew Gwynne, who is doing an airplane wing walk in her honor this May!

In Loving Memory

Ways to Give – Your Support Matters

Charity Miles | RoundUp App | Facebook Fundraisers | Donate | Set up a Recurring Donation

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