Amazing Advocacy this Rare Disease Day

Our community really showed up to participate in Rare Disease Day this year! Many of you filled in coloring pages, completed crosswords, wore stripes, submitted photos for our photo contest, and donated. Every action had an impact! Thank you!

  • The A Cure for Violet video campaign raised over $35K for Rare Disease Day! Thank you Violet and family, and thank you to everyone who donated!
  • Thanks to the Rare in Times Square campaign run by Beyond the Diagnosis, the Jumbotron in Times Square featured Sweet Iris to spread awareness for GM1 and Rare Disease Day.
  • The winner of the photo contest was Debora Corso. Thank you Debora for a beautiful photo, and thank you to everyone else who submitted entries. There were many touching photos to choose from. 

The 2023 WORLDSymposium

Thank you to everyone who presented at WORLDSymposium, and thank you to all the companies, researchers, doctors and professionals who have or are presently working on GM1. 

At this year’s WORLDSymposium, Cure GM1 founder Christine Waggoner received the WORLD 2023 PAL Award for her excellence in GM1 patient advocacy, and TEGA Therapeutics presented on the development and validation of enzyme replacement therapy for GM1 gangliosidosis.

Other GM1-related presentations include:

  • Passage Bio presented updated interim safety, biomarker, and efficacy data from Imagine-1: A phase 1/2 open-label, multicenter study to assess the safety, tolerability, and efficacy of a single dose, intra-cisterna magna (ICM) administration of PBGM01 in subjects with type I (early onset) and type IIA (late onset) infantile GM1 gangliosidosis (GM1). Read the press release and watch CGTLive’s interview with Dr. Samiah Al-Zaidy, VP of clinical development and lead on the GM1 program.  Passage Bio also announced a new cohort at a higher dose will be enrolled in the second half of 2023.
  • St. Jude Children’s Research Hospital presented preclinical enzyme replacement therapy with a recombinant β-galactosidase lectin fusion for CNS delivery and treatment of GM1-gangliosidosis.
  • Codexis presented an engineered β-galactosidase with improved stability and cross-correction for the potential treatment of GM1 gangliosidosis via AAV gene therapy.
  • Codexis presented In vitro modeling of GM1 Gangliosidosis using iPSC-derived cellular and organoid CNS models.

Coming Up

March is Developmental Disabilities Awareness month.

Raise awareness so we can better include and connect with people with developmental disabilities in all aspects of our communities, as well as lift the barriers they face in using their local amenities.

March Caregiver Support Group

The next caregiver support group with Dr. Al will take place on March 28th, 2023 at 12:00 PM Pacific Time. All caregivers for those with GM1 are welcome and encouraged to come. Email to be added to the invitation.

Proclamations Needed

GM1 Day will be here soon – May 23rd! Our goal is to have as many proclamations granted as possible to make the day truly official.

In 2022 we got proclamations in CO, AZ, IA, and MI. This year, proclamations are in the works in CT, NJ, MA, VA, UT, TX, and NC. If your state is not listed, please let us know if you can request a proclamation. Email us at

Cure Kinley & Kennedy Annual Golf Tournament

Registration for the 3rd Annual Cure Kinley & Kennedy Golf Tournament benefiting Cure GM1 is open! If you live in or near Westminster, Colorado you can easily join them for a fun day of golfing to support a wonderful cause.

Face Off for Marley Charity Hockey Game

If you’re in or near Kalamazoo, you are invited to attend A Cure for Marley is hosting Face Off for Marley Charity Hockey Game on April 8th at 11:30 AM.

Support Cure GM1

Flower Power

Now is the perfect time to order spring bulbs for your garden. When you get them from Flower Power, you also support Cure GM1. There are also seeds, ornamental plants, and flowers for bees and hummingbirds.

Order in March

See’s Easter chocolates are available now through the end of March. Ordering is only available through the end of March. Make sure to order soon to get your supply for Easter!

RoundUP at Walmart in March

Between March 1 and March 31, 2023, Walmart is matching customer donations 2:1 when you round up your purchases. Make sure to donate to Cure GM1 while shopping at Walmart online.

Happy Birthday!

  • Frieda – Feb 3
  • Tiffany – Feb 4
  • Camden – Feb 5
  • Iris – Feb 19
  • Kennedy – Feb 19
  • Aidan – Feb 23
  • Paxton – Feb

Ways to Give – Your Support Matters

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

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