2024 Cure GM1 Virtual Conference

We are excited to start planning our annual conference for 2024! Please save the date and make a plan to join us. In 2024, we will have a virtual conference and we will plan to hold hybrid and virtual conferences in the future. We truly look forward to this important event which will provide the latest updates on research, trials, and more!

Iris’s Sweet 16 Birthday and Video

$27K Raised in Honor of Iris

Thank you to everyone who has donated in honor of Iris and her Sweet 16! For GM1 families, birthdays can be bittersweet because the passage of time is worrisome. Knowing we are working towards a brighter future helps to cope with navigating this devastating rare disease journey.

GM1 Community Features

Throughout February, we shared special new features of community members. Please take a few minutes to read these extremely personal and powerful accounts of living with GM1.

  • Marley has late-infantile Type 2a GM1
  • Jessica has late-onset Type 3 GM1
  • Carter shares his perspective as a sibling of Iris who has Type 2b juvenile GM1

“It’s unimaginably difficult navigating the roads of a rare, painful, degenerative, fatal disease and the only way we can make it easier is through support. Donations for research will improve the medical community’s ability to properly treat and guide the patients.”

Jessica, adult with Type 3 GM1

Goodbye and Thank you

Our communications and outreach coordinator, Caitlin Dwyer, has moved on to a new endeavor. We thank her for her work in making our community organized, thoughtful, and vibrant. She writes “The advocacy you do is powerful. It was lovely to meet and get to know you all. I believe in this cause and hope for the best for all of you.”

World Symposium

Our founder and president, Christine Waggoner attended the WORLD Symposium meeting in San Diego to represent Cure GM1.  Every day was filled from dawn to evening with meetings and talks.  This conference is an excellent opportunity to learn the latest news and to connect with researchers, physicians, stakeholders, patient advocates and biotech companies.  The WORLD Symposium is an incredibly important meeting. We encourage interested patient families to attend in the future and to take advantage of free admission.  The more visibility and more people advocating for GM1 will help draw interest and continued progress.

At the conference, there was a significant uptick in presentations and posters pertaining to GM1 this year.  It is hopeful to see these presentations and to meet with the teams.

  • Arunabha Ghosh, St. Mary’s Hospital, Manchester, United Kingdom: Safety and preliminary efficacy of LYS-GM101 gene therapy in patients with GM1 gangliosidosis: Results of a phase I/II open-label clinical trial
  • Precilla D’Souza, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, United States: Intravenous delivery of AAV9-GLB1 gene therapy for GM1 gangliosidosis: An interim analysis
  • Maria Acosta, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, United States: Gains in neuronal tracks in GM1 gangliosidosis patients following intravenous gene therapy. Differential tractography a robust outcome measure for neurodegenerative disease
  • Samiah Al-Zaidy, PassageBio: Intra-cisterna magna administration of AAV gene therapy for neurodegenerative disorders

WORLD Symposium Posters

All the research would not be possible without GM1 community participants. Thank you to everyone who helps advance all these projects.

  • Pamela Kell, Application of a pentasaccharide biomarker to access treatment efficacy of gene therapy for GM1 gangliosidosis

NIH Posters

  • Maria Acosta, Comparing GM1 gangliosidosis patients treated and untreated with gene therapy: Longitudinal white matter changes in GM1 using differential tractography
  • Precilla D’Souza, Intravenous delivery of AAV9-GLB1 gene therapy for GM1 gangliosidosis: An interim analysis

Lysogene Poster

  • Arunabha Ghosh, Safety and preliminary efficacy of LYS-GM101 gene therapy in patients with GM1 gangliosidosis: Results of a phase I/II open-label clinical trial

Azaforos Posters

Thank you to all participants in the Azafaros natural history study!

  • Roberto Giugliani, A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Baseline clinical data
  • Roberto Giugliani, A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): patients’ and caregivers’ assessments
  •  Roberto Giugliani, A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Evaluation of different assessment
  • Kyle Landskroner, Nizubaglustat regulates GM1 ganglioside neuronal health in a human brain organoid model of GM1 gangliosidosis
  • Renata Medinaceli, Quintela: Use of wearable sensor technology to identify digital biomarkers for monitoring gait parameters in children and adolescents with GM1 and GM2 gangliosidoses

Sanofi Poster

A special thank you to the caregivers who participated in the research study that supported this poster.  The study is now bearing fruit and the results are being shown at scientific conferences throughout the year.  

  • Daisy Ng-Mak, Motor function limitations and impacts among patients with GM1 and GM2 gangliosidoses in the United States: A qualitative study

Cure GM1 Meets Novel Pharma at WORLD

At the WORLD Symposium, it was a pleasure to meet with a members of a new startup in Korea. Novel Pharma is developing an intracerebroventricular(ICV) enzyme replacement therapy (ERT) for GM1.  Novel Pharma is a seasoned team who worked on an ERT called Hunterase for another lysosomal disease called MPS II / Hunter syndrome.  Hunterase is approved in Japan and Korea.  We will share more information as news becomes available.

Reagan-Udall Meeting Supports Accelerated Approval Based on Biomarkers for rare Diseases

A fascinating and important meeting was organized with respect to qualifying heparan sulfate as a biomarker for Mucopolysarcharidoses / MPS diseases as a basis for accelerated approval. If you missed this meeting, see the slides and agenda.

Why is this relevant to GM1?

Like GM1, MPS conditions are rare neurodegenerative lysosomal diseases that primarily impact children.  GM1 gangliosidosis has biomarkers which are hallmarks of the disease, e.g., beta-galactosidase, GM1 ganglioside, and other well-known biomarkers, e.g., keratan sulfate and glycosaminoglycans.  Given that GM1 gangliosidosis is a progressive disease, it can be difficult to conduct clinical trials due to heterogeneity and significant neurodegeneration.  Therefore, approval based on biomarkers could help encourage drug development and approval of possible treatments for GM1.

There were powerful testimonies and quotes from the meeting.

“All children deserve the chance to live. We do not need your sympathy; we do not need your empathy; we need action from our FDA partners.”

Mark Dant, Father, Advocate and Executive Director for The Ryan Foundation

“We all see the need to lean into biomarkers in order to lean into accelerated approval for rare diseases, otherwise we could leave patients behind and put programs at risk that could help patients…We encourage people to engage with us on biomarker development and accelerated approval, which is going to be the norm for a lot of initial approvals especially in gene therapy.”

Dr. Peter Marks, Center for Biologics Evaluation and Research, FDA

Rare Disease Month

A very special thank you to Mix Talent for featuring our founder and president Christine Waggoner as “The Crusader” for Rare Disease Month. Please read more here.

Rare Disease Day Proclamation Honoring Violet Law

California Senator Padilla invited David Law’s family to Sacramento to honoring Violet’s memory on Rare Disease Day by issuing a proclamation again in honor of GM1 Day in 2024. You can read the Long Beach Post article here.

Rare Disease Day Photo Contest

Thank you so much to everyone who entered our Rare Disease Day photo contest. We are pleased to announce that Marley’s mom’s photo is this year’s winner. All the photos are beautiful and we still share each and every one of the entries. Thank you!

Preparation for GM1 Awareness Day 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. Make a plan to spread awareness or hold an event.

  • 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

Ways to Help

Join us!

The reality is that if we must continue to advocate and bring more resources to our mission and fight in order to make a meaningful difference. There has already been a great deal of change, but persistence is key. Do you have questions about how to help? Would you like to chat? Do you want to volunteer? There are ways to help big and small. Thank you.

Minted Discount

Minted.com, the online marketplace of premium design goods created by independent artists and designers, is offering a Year-Round discount to benefit Cure GM1! The discount code is FUNDRAISECGM1 for 20% off + free shipping, and 15% will be donated back to the Cure GM1 Foundation!

RaiseRight

Support Cure GM1 With Every Day 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.
  • RaiseRight purchases can support Cure GM1 through major retailers such as Apple, Starbucks, Gap, and Amazon. Airline tickets, groceries, and gas cards can all support Cure GM1.

Happy March Birthdays!

  • Marley – March 9
  • Armand – March 13
  • Milan – March 17
  • Jenny – March 20
  • Keeton – March 21
  • Toby – March 21
  • Mira – March 28

In Loving Memory

We extend our deepest condolences to Aryana, Luis Gabriel, and Valentine’s families whose beloved children passed away this February.

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.