Cure SCG

05/28/2026

05/07/2026

Every child deserves the chance to run, play, dream, and grow without limits.

But for children living with Limb-girdle muscular dystrophy type 2C, everyday life can become increasingly difficult as muscle weakness progresses over time.

Today, we share the stories of three incredible children —Jayla, Donia and Samy — whose journeys are filled with courage, resilience, and hope.

They are more than a diagnosis.
More than their condition.
They are children with dreams, joy, and futures worth fighting for.

Over the coming weeks, we will share their stories to raise awareness and help bring hope to families affected by LGMD 2C.

Every donation helps move research one step closer to better treatments — and one day, a cure.
You can make it feel like a button by formatting like this:

👉 READ THEIR STORIES
https://www.curescg.org/our-stories

👉 SUPPORT RESEARCH & DONATE
https://www.curescg.org/donate

04/23/2026

Art from the Heart: A Stroke of Gratitude 🎨❤️
For the past few months, the incredible team at has been so much more than a medical staff; they have been Sara’s source of strength, comfort, and care.
Sara wanted to find a way to truly say “thank you” for the beautiful way they looked after her, and she knew she couldn’t do it better than through her art. She poured her heart into these paintings as a gift for the team, hoping it serves as a lasting reminder of the incredible impact they have on their patients every single day.
On behalf of CureSCG, we are deeply grateful to the UF team for their kindness, expertise, and for walking this journey with Sara. 🏥✨

04/01/2026

Two months after gene therapy, watching Sara walk feels different.

Before treatment, she used to fall multiple times a day. Some of those falls were really hard… and honestly, very scary. As a parent, you never get used to that feeling.

Now, two months later, I can’t even remember the last time she fell.

For us, this is life changing.
These are the moments that matter the most… the ones you don’t always see captured in clinical endpoints or measurements.
But as parents, we feel it every single day.

03/09/2026

Hope for families living with LGMD 💚

Very encouraging early results from the ATA-200 clinical trial for LGMD R5 (gamma sarcoglycanopathy) were presented during the MDA Clinical and Scientific Conference, bringing renewed hope to families affected by this rare neuromuscular disease.

🔬 Key findings include
• >90% SGCG expression in muscle fibers at 6 months
• Sustained reductions in CPK and transaminases at 9 months
• Encouraging functional improvements in ambulatory patients
• No serious adverse events reported in the four treated children

Progress in rare diseases is the result of a collective effort with patients, families, researchers, and scientists working together toward better treatment options.

For families living with sarcoglycanopathy, every step forward in research brings renewed hope.

For more information, read the full press release
https://urls.fr/eL0ZLn

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03/03/2026

This moment means hope 💚
Supporting the ATA-200 clinical program for LGMD 2C reminds us why we keep going. The dosing of a fourth patient is more than science; it’s progress for families who have been waiting for answers.

Rare disease progress happens when families, researchers, clinicians, and advocates move forward together. We’re deeply grateful to everyone helping make this possible.

Time is muscle. Patients can’t wait, and every milestone matters 💪
We’ll keep pushing so more children and families can have hope too.

02/28/2026

On behalf of LGMD CAP
Help shape better cardiac care for people living with LGMD

We are launching an international patient survey to capture the lived experiences of up to 140 individuals across multiple LGMD subtypes. The goal is to better understand real-world experiences related to access to cardiac care, treatment satisfaction, and daily quality of life, and to explore how these experiences differ across countries.

By listening directly to patients and families, this survey aims to identify meaningful patterns and unmet needs that are often missed in clinical settings alone.

The findings will be included in a scientific publication and presented at the ENMC workshop on cardiac care in November 2026. Bringing the patient voice directly into this expert forum will help strengthen clinical communication, inform future patient engagement strategies, and support more patient-centered approaches to care.

The results will also contribute to the development of a dedicated masterclass for cardiologists treating LGMD patients, created in partnership with Treat NMD, to support improved understanding and management of cardiac involvement in LGMD.

Your data is safe and protected.
All responses will be fully anonymized prior to analysis and securely stored by Treat NMD.

If you are living with LGMD or caring for someone who is, we encourage you to share your experience and help shape the future of cardiac care for the LGMD community.

👉 To take the survey; send an email to [email protected]

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02/28/2026

Rare Disease Day 💚

Our journey with rare disease began only three years ago.
Three years of learning a new language we never expected to learn; medical terms, clinical trials, uncertainty, and hope.

In this short time, we advocated. We traveled across countries. We sat in medical conferences that, at first, felt overwhelming and impossible to understand. Slowly, we learned. We asked questions. We kept showing up.

We partnered with researchers and health institutions, working to help move science forward. But the strongest connection we found was not in conference rooms; it was with other parents and children.

We understand each other without explanation.
We feel what others feel.
Our children connect instantly, as if they already know they are not alone.
We hold each other up when the weight becomes too heavy.

Some families have been fighting for decades. Their strength lights the path for families like ours. Their advocacy made space for progress, for research, for hope.

To every parent raising a child with a rare disease: you are the momentum behind change. You are the voice science listens to. You are the reason progress continues.

Today we raise awareness for rare diseases and for Limb-Girdle Muscular Dystrophy (LGMD).
Awareness leads to research.
Research leads to treatments.
And treatments lead to hope.

No family should walk this path alone.
Together, we move forward.
Together, we keep going.
Time is muscle.

02/20/2026

Ramadan is a season of mercy, generosity, and hope 🌙💚

For families living with Limb-Girdle Muscular Dystrophy, hope means research moving forward and treatments that cannot wait.

At CureSCG, every donation helps accelerate gene therapy research and brings us closer to real options for children and families affected by sarcoglycanopathy.

This Ramadan, your support can make a meaningful difference and help ensure patients are not left waiting.

Donate, share, and be part of the progress.
🔗 Donation link in bio

01/29/2026

It was wonderful to see Joe and Peter from the and spend time together. We are deeply thankful for their continued hard work and dedication in advancing the first ever LGMD2C clinical trial, which has brought hope to so many families in our community.

What made this moment especially meaningful was seeing children with the same condition connect with one another. These moments of understanding, belonging, and shared experience matter just as much as the science. They remind us why community is so important for our kids and our families.

As a nonprofit supporting research and patient advocacy, we are grateful to stand alongside those working tirelessly to move LGMD research forward and to build a stronger, more connected community for our children.

Thank you, Dion Foundation, for all that you do. 💚