Thank You
Thank you for your support in 2022! Our mission to help develop effective treatment options and therapies for GM1 can continue into 2023 because of community members like you. This work takes every kind of effort and it takes all of us. All means of support, big or small, have advanced our cause. Thank you for reading our emails, liking our posts, advocating, and donating.
Coming up in 2023
Dr. Al Caregiver Support Session
Dr. Al Freedman will be leading a support group for GM1 caregivers on January 17th from 3:00 – 4:00 PM PST. Join a private group session and connect with others who share your experiences. Reserve your spot today by emailing info@curegm1.org.
WORLDSymposia Registration
The WORLDSymposium, a conference dedicated to research and advances in lysosomal diseases, takes place from February 22nd – 26th, 2023 in Orlando, Florida. Registration is FREE for GM1 families. Christine Waggoner, our President and Founder is this year’s recipient of the Patient Advocacy Leadership Award and we are honored to receive this important recognition. Join us at the conference!
Rare Disease Day 2023
Rare Disease Day is on February 28th, 2023. Read our blog post on nine ways you can observe Rare Disease Day this year.
There are three events observing RDD:
- Rare Disease Day At NIH 2023 is on Monday, February 28th at the Natcher Conference Center in Bethesda, Maryland.
- FDA Rare Disease Day Virtual Public Meeting is on Monday, February 27th from 9:00 am – 4:45 pm EST.
- EveryLife Foundation’s Rare Disease Week on Capital Hill will open registration on Thursday, January 4th.
AllStripes has characterized the diagnostic journey of patients with GM1 gangliosidosis using real-world data in a visual guide. To everyone who contributed to the AllStripes research, thank you. Interested in participating? You can join AllStripes here.
On December 14th, Passage Bio announced positive interim clinical data from the first six patients with GM1 gangliosidosis in their Imagine-1 Study See the interim safety, biomarker, and efficacy results from cohorts 1-3.
Read a recent article with respect to a Lysogene preclinical study and their AAVrh10 vector. This preclinical research preceded the LYS-GM101 human clinical trial, which has dosed five patients to date.
News
Caregiver Preferences
The results of Cure GM1’s Caregiver Preferences study are available to read in the first ever publication of its kind! Caregiver views and experiences should inform and drive drug development. This was a multi-year collaborative effort, and we are proud to have this publication in the American Journal of Medical Genetics. This paper will be part of the literature of GM1 and read by many geneticists and medical professionals. Thank you to all our collaborators and sponsors for making this work possible!
Marley, a GM1 patient in our community, was included in the article, Rare disease therapeutics: The future of medical genetics in a changing landscape. The article includes Marley’s experience of being ineligible for a clinical trial due to age criteria. It is our hope that ultimately with a treatment approval, access to treatment will be greatly increased.
Include Cure GM1 in your New Years Resolutions
Support Cure GM1 with every mile you run through Charity Miles. It’s super easy to get set up with their app, and will give you another source of motivation to get your miles in. Indoor and outdoor exercise can both be tracked.
Round up your purchases online and in stores with the RoundUp App. They make it easy to link your credit card so every purchase gives you the option to round up to the next dollar. Every little bit helps our cause!
Advocacy Success
Congress passed a year-end legislative package, H.R. 2617, in favor of those with rare diseases. They voted to strengthen the FDA’s approval pathway, renew several programs through 2027, and insure increased presence of underrepresented populations in clinical trials.
You can take action to support GM1 families. The proposed legislation, The Right to Build Families Act of 2022, would protect access to assisted reproductive technologies, including the use of IVF to conceive. Call or send a message to your representative today. Let them know assisted reproductive technologies and IVF are important to you.
Ways to Give – Your Support Matters
Charity Miles | Facebook Fundraisers | Donate | Set up a Recurring Donation
Visit our Take Action page for more ways to support our community