Rare Disease Day

This Rare Disease Day – February 29th – will be a powerful day of advocacy for Cure GM1. Help us to spread awareness about GM1 gangliosidosis. Simple, easy ways you can take part include:

Coming Up

Rare Disease Day at the NIH

On Thursday, Feb. 29, from 9 am to 5 pm ET, NIH will hold the free event, “Rare Disease Day at NIH” at the Natcher Conference Center in Bethesda, MD. A livestream will also be available. The event will include panel discussions, rare diseases stories, in-person exhibitors, scientific posters, and an art exhibition. Register here.

FDA to Hold Rare Disease Day 2024 Public Meeting

On March 1st, 2024 from 9am to 4:30 PM, the FDA will hold a virtual public meeting in observance of Rare Disease Day. There will be multiple panel discussions and an opportunity for continuing education credentials for healthcare professionals. You are invited to provide perspectives on the public docket. Register here.

Tell Your Story Webinar

Global Genes is hosting the Tell Your Story Webinar on February 14th. It will teach and empower attendees to tell their story in video. Register here.

Qualifying Biomarkers Workshop

In the “Qualifying Biomarkers to Support Rare Disease Regulatory Pathways,” you will learn about how the FDA assesses possible treatments for lysosomal storage diseases. This is a day-long hybrid public workshop that takes place on February 21st from 10am – 4pm ET. It will feature heparan sulfate in neuronopathic lysosomal storage diseases as a case example. Register here.

GM1 Day is in May

GM1 Day Proclamations – If you live in one of these states, you can help us advocate this GM1 Day by asking your state to proclaim GM1 Day an official day of observance. Email us at info@curegm1.org if you can help.

  • Alabama
  • Arkansas
  • Delaware
  • Georgia
  • Hawaii
  • Indiana
  • Kansas
  • Kentucky
  • Louisiana
  • Maryland
  • Maine
  • Missouri
  • Mississippi
  • North Dakota
  • Nebraska
  • New Hampshire
  • New Mexico
  • Nevada
  • Oregon
  • Pennsylvania
  • Rhode Island
  • South Dakota
  • Tennessee
  • Vermont
  • West Virginia
  • Wyoming


Small Molecule Update

Gain Therapeutics has “published data in PLOS ONE on our allosteric small molecule modulators of the GLB1 enzyme showing significantly restored β-Gal function and reduced intracellular toxic substrates as part of our program for GM1 gangliosidosis.” Read the press release here

PRONTO Study Update

Azafaros is to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the annual WORLDSymposium. Read the press release here.

Get Paid to Take Rare Disease Surveys

At Rare Patient Voice you can sign up to take paid surveys, phone interviews, online bulletin boards, focus groups, clinical trials, and more at a generous $120 per hour. For each person who signs up, Cure GM1 will receive $10. This research helps progress research into treatments and therapies for GM1. Sign up today!

Support Cure GM1

Contribute with Purchases
  • Powerful Impact Possible with RaiseRight – Those who use RaiseRight for their everyday shopping needs can earn over $1,000 a year for Cure GM1. You don’t need to fundraise or expend time or spend extra funds. It’s easy to use – find out how here.
  • Send your Valentine a bouquet – With the Crayola Flowers program, all flowers you buy will help support Cure GM1. Order a bouquet here.
  • Gift Chocolates to your Valentine  – Part of your purchase of Sees Valentine’s chocolates will go toward Cure GM1. Order some for your loved ones today
Host a Fundraiser

With a new year is a new opportunity to work towards continued progress. Consider hosting birthday and special occasion fundraisers, which are easy and meaningful.

Include Cure GM1 in Your Will 

Join a strong contingent of supporters who are making a lasting impact in the fight against GM1. Consider supporting Cure GM1 through planned giving, and help us continue our vital work of developing effective treatments and therapies for GM1, by adding us to your will.  

Happy January Birthdays!

  • Vittoria Anne – Jan 2
  • Darian – Jan 6
  • Lucas – Jan 9
  • Florence – Jan 30

In Loving Memory

Ways to Give – Your Support Matters

RaiseRight | iGive | Charity Miles | Facebook Fundraisers | Donate 

 Set up a Recurring Donation

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