We hold space for the parents in our GM1 community who have said goodbye to a child far too soon. Every laugh, every milestone, every quiet moment mattered, and your child's memory lives on in all of us. To every GM1 family carrying this loss: you are not alone. Today and always, we remember with…

“I realize how the disease gradually takes away so many abilities. It is like slow torture, without apparent pain but with deep hurt, always present, ready to surface unexpectedly.” GM1 Parent Living with GM1 gangliosidosis is a kind of grief that does not arrive all at once. It comes in pieces. In…

They may not say they're hot. GM1 gangliosidosis affects the nervous system. Many of our kiddos cannot reliably feel when they are overheating, dehydrated, or uncomfortable in the sun. Some cannot tell us in the usual ways. #CureGM1 #GM1 #RareDisease #SummerSafety #SensoryProcessing #SpecialNeedsP…

July is Disability Pride Month. We celebrate every ability, honor every journey, and stand with every family in the GM1 community navigating a rare disease with extraordinary grace. You are seen. You are valued. You matter. #DisabilityPrideMonth #CureGM1

Today, he plays. Tomorrow, he deserves the chance to keep playing. GM1 gangliosidosis is rare, but the love these children inspire is anything but. Every smile you see in this photo is a reason to keep going. Every laugh is a reminder of what we're fighting for. And every step forward in research i…

Join us in welcoming Cleopatra Frazier as our coordinator for the 2026 International GM1 Community Conference! Cleopatra will be your point of contact for this year’s *virtual* event Friday, Oct. 2. Click the link in bio to register now! Registration is FREE for GM1 families.

Today is International Newborn Screening Day, and for families facing GM1 gangliosidosis, early detection means everything. By the time the first symptoms appear, irreversible neurological damage has often already begun. The science is ready. LEAR MORE: curegm1.org/nbs-gm1 #NewbornScreening #G…

Please join us in wishing Kinley a wonderful birthday today! Drop your birthday wishes below! #curegm1 #gm1gangliosidosis

Caregiving carries weight. The emotional load of rare disease caregiving is real. Sleep interrupted for years. Appointments that never end. Grief that lives alongside love. Many GM1 caregivers experience symptoms of post traumatic stress, and most have never been told that what they are feeling has…

Progress in biotech takes more than science alone. It takes advocates who raise awareness, build partnerships, and keep patient needs at the heart of every conversation. We were proud to attend BIO in San Diego this week. #GM1 #RareDisease #PatientAdvocacy #Biotech

A common cold can become life threatening for children living with GM1. Help fund enzyme replacement research at curegm1.org/ert-project #GM1 #CureGM1 #EnzymeReplacementTherapy #RareDisease #ChildHealth #FundResearch

Today we remember baby Ada, whose life was filled with love and whose loss reminds us why awareness and research matter so urgently. For Ada and every child affected by GM1, we keep pushing for progress. #GM1 #RareDiseaseAwareness #InfantileGM1

Caregiver Check-In, Week 4: you are doing enough. The love you give does not need a perfect day to be real. On the hard days. On the quiet days. On the days you barely got through. It still counts. You are doing enough. Send this to a caregiver who needs to hear it today. #PTSDAwarenessMonth #Careg…

Today, we honor fathers whose love, strength, and dedication shine brightly within the GM1 community. Thank you for your strength, your kindness, and the difference you make every single day. #CureGM1 #RareDiseaseAwareness #community

Today we honor the dads in the GM1 community. Thank you for your love, strength, advocacy, and steady presence. Your families and this community are stronger because of you. #curegm1 #fathersday #charity

Our thoughts are with Armand's family, friends, classmates, and all who are grieving this profound loss. May the memories he created, the laughter he shared, and the love he gave continue to shine through those whose lives he touched. Forever remembered. Forever loved. #RememberingArmand #InLovingM…

Gustavo in the lap of his father Diego, who takes care of his son with complete love and dedication. #curegm1 #fathersday #caregiving

Tomorrow is Father’s Day 💙 Honor a GM1 dad by sharing a photo, memory, or message on June 21st. Here’s to the dads, granddads, stepdads, and father figures who show up with quiet strength and steady love. #CureGM1 #GM1Community #FathersDay #GM1Dads

This week, we continue to honor the fathers in our GM1 community. Join us in wishing them love and gratitude. "Connor is the kind of dad who quietly carries the weight of the world for his family. ... He has never wavered in his love or commitment to our son. What makes him such a wonderful dad is…

“Mila enjoys every second with her daddy. They laugh, cuddle and have lots of fun being silly together.” Join us in acknowledging all of the fathers who work hard and love softly. We are so grateful you are here! #gm1 #curegm1 #gm1dads

"Super Dad is an understatement of how much Kyle does on a daily basis. He is a caregiver, a friend and the best dad his girls could ask for! Thank you for taking such good care of all of us.“ - Kinley & Kennedy’s Mom. Share what makes your partner so special below!

“Every girl deserves a hero. Nella got the very best one." Let's hear it for the dads! Join us in celebrating all of the wonderful fathers in our GM1 community. Drop your Father's Day wish below. #gm1 #curegm1

"Ray is the most patient and sweetest dad. Tiffany’s face lights up every time she sees him." - Tiffany's Mom. Our countdown to Father's Day continues! What makes your partner so special? Share below so we can celebrate him with you.

This Father's Day week, we recognize all of the men stepping up to help guide and support their families impacted by GM1. We see you, we honor you, and we wish you a wonderful weekend filled with love. Drop your Father's Day wishes below! #gm1 #curegm1

This week, we begin our countdown to Father's Day. Please join us in sending love to every father in our Cure GM1 community. "A father's job is to protect and comfort. Even when the world was heavy, you created a sanctuary of love and peace for Daxton." - Daxton’s Mom #curegm1 #gm1

Caregiver Check-In, Week 3: asking is strength. Asking for help is not a failure of caregiving. It is caregiving. A meal. A ride. A listening ear. A break in the afternoon. Let people in. Name one thing you could ask for this week. Out loud, or in the comments below. Practicing the ask is part of t…

☀️ Connect, share, and build community with other GM1 families! Join us for our June Community Chat on June 25 at 2:30 p.m. PDT / 5:30 p.m. EDT for a casual conversation and an opportunity to connect with others who understand the GM1 journey. Scan the QR code to join. We look forward to seeing yo…

Every drop. Every sample. Every step forward. World Blood Donor Day celebrates the people who give so that others can live. The same spirit drives rare disease research. Participation, donation, and partnership are what move medicine forward for conditions like GM1 gangliosidosis. If you have ever…

A fact every GM1 family should know. In a published natural history study of GM1 gangliosidosis, respiratory complications, most often aspiration pneumonia, were the most common cause of death, regardless of which treatments or palliative therapies the patients were using. Citation: Jarnes Utz JR,…

Today we send belated heavenly birthday wishes to sweet Violet, who is gone far too soon. We hold you and your family in our thoughts today and always. #gm1 #curegm1

Today, she dances. And every twirl, every spin, every giggle is a gift. Children with GM1 gangliosidosis are racing against a clock most of us will never have to think about. But thanks to the families, scientists, and supporters in this community, that clock is no longer the only story being writt…

“I wish people understood that rare doesn’t mean less real.” GM1 Parent Rare disease is invisible to most of the world, but it is not invisible to the families living it. Behind every statistic is a child, a parent, a household reorganized around love and care. Help us make GM1 visible. Like, sh…

Today, we celebrate the birthdays of Isabella and Julia. Their light and joy are contagious. We wish them a happy day filled with love and family. Drop your birthday wishes for the girls below!

Honor Dad with a gift that makes a difference. Every Cure GM1 t-shirt helps raise awareness and support the work moving GM1 research forward. SHOP: curegm1.org/gm1-store #CureGM1 #GM1Awareness #FathersDay

Exciting new publication by Azafaros accepted to a peer-reviewed medical journal of the RAINBOW study. Topline results from the study announced in 2024 demonstrated that nizubaglustat was safe and well tolerated, with encouraging signs of efficacy. https://tinyurl.com/4fmafuw5 #curegm1 #raredise…

Caregiver Check-In, Week 2: find your people. Connection eases the weight. Send the text. Reply to the message. Sit on a call with another GM1 family. You do not have to explain everything to be understood here. Reach out to one person in this community this week. A note, a voice message, a wave. I…

Today we honor a precious baby whose life was far too short. GM1 stole moments that should have lasted a lifetime. We remember, we grieve, and we keep fighting for families. #curegm1 #raredisease #sweetangels #childloss

Rare disease language can be confusing. Anti-GM1 antibodies and GM1 gangliosidosis may sound similar, but they refer to very different medical issues. READ MORE: https://tinyurl.com/4rewswud #curegm1 #raredisease #medicine #genetics #neurology

Do It For GM1 is bringing us closer to a world where children with GM1 can live longer, healthier lives. Choose your way to make a difference, rally your community, and help accelerate critical research, support families, and advance promising treatments like enzyme replacement therapy for GM1 gang…

Happy Birthday, Oliver! From all of us at Cure GM1, we’re sending you love, hugs, and the biggest birthday wishes. We’re so grateful to celebrate you today and the incredible person you are. Wishing you a day as wonderful as you are! #curegm1 #birthdays #charity

Caregiver Check-In, Week 1: rest counts. Rest is not a reward you earn after everything is done. It is part of how you keep going. Five quiet minutes count. A nap counts. Asking for an hour counts. This week, tell us one way you will rest. Even if it feels small. Someone in this community needs to…

"In the smallest footprints, you left the biggest mark on our hearts. Until we meet again, sweet angel.” -unknown #curegm1 #sweetangels #childloss #love

Our June 2026 Cure GM1 Catalyst is here and it's a celebration! GM1 Day 2026 was one for the books. This month's newsletter is packed with: GM1 research highlights from the ASGCT Annual Meeting The story of three brothers: Joey, Oliver and Dominic "Clara's World" heading to Children's National Medi…

To the GM1 parents: Thank you for the advocacy. The late nights. The gentle care. The love you give every single day. Your strength is the heartbeat of this community, and the reason research moves forward. Today, on Global Day of Parents, we are thinking of every Cure GM1 family. The parents who l…

Today is National Smile Day, and if there is one thing this community knows, it's that smiles are sacred. In the world of GM1, a smile is so much more than an expression. It's a whole story. The flicker of joy when a favorite song comes on. The grin that says "I see you, I love you." The quiet, b…

If you have a celebration coming up, would you consider dedicating it to Cure GM1? We will be cheering you on, sending resources to make it easy, and lifting up your story. Learn how to start a fundraiser at curegm1.org/take-action 🎂 Every candle. Every wish. Every shared moment. They can all be…

Why does advocacy matter? Raising awareness brings hope. It increases research funding. It helps families feel seen and supported. Every post shared moves us closer to a cure for GM1 gangliosidosis. Share this with one person who has never heard of GM1. That is how rare disease awareness grows. cur…

“We are diagnosing your son with GM1.” “I felt numb.” GM1 Parent Every GM1 family remembers the room. The chair. The way the words landed. Diagnosis day does not end at the door of the appointment. It echoes for years. To anyone in this season right now: you are not alone. Cure GM1 is here, and…

Recognizing the early warning signs of GM1 gangliosidosis can save precious time. Watch for developmental delay or regression, muscle weakness and poor motor control, and neurological symptoms like clumsiness or speech issues. Early signs are often subtle and easy to miss, especially in a first chi…