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Together for
Limb-Girdle
Foundation · Fighting LGMD

Two young men. Two families. A progressive muscle disease. No treatment. A potential gene therapy. Scientists who have spent years getting it this far. And now a financial gap stands between the laboratory and a first clinical trial.

For Floris and Tobias, and others like them, it is essential that Phase 1 starts as soon as possible. Through your donation, we can accelerate the path from care to a real cure, changing the course of their future.

Support our mission Our Story
Floris and Tobias smiling outdoors

Paving the way fromcare to cure

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Patients worldwide
>100K
people living with LGMD-R1/2A
Most common form
#1 LGMD 2A
Most frequent subtype worldwide
Disease progression
10–25 yrs
until loss of walking ability
Approved treatments
0
No disease-modifying therapy exists
€10M The Opportunity

A potential gene therapy is ready for the next step

More than a decade of research has brought ATA-300 to the threshold of clinical development. Funding is needed to prepare the first Phase 1 clinical trial for people with Limb-Girdle 2A/R1, manufacture the therapy, and treat the first six patients. Results from earlier successful trials for other LGMD subtypes have provided important knowledge and data for this development. In the same way, the LGMD 2A trial can generate new insights that also contribute to developing treatments for other LGMD subtypes. Our mission is to help make that next step possible.

Help fund the path to Phase 1 Gene therapy successes
Research lab Gene therapy research
Who we are

A foundation built by parents, for our children and others like them

The Together for Limb-Girdle Foundation was founded by the parents of Tobias (18) and Floris (21), two young men from the Netherlands living with Limb-Girdle Muscular Dystrophy type 2A/R1 (LGMD2A/R1). What began years ago as a personal quest for answers grew into a shared mission: to accelerate research and bring effective treatments for this progressive muscle disease closer to reality.

From our own experience, we know that waiting is not an option. LGMD is a disease that progresses every day. That is why we believe there is no time to lose. Speed is essential, in research, in collaboration, and in the development of solutions.

Because LGMD is a rare disease, pharmaceutical companies do not automatically prioritize the development of treatments. That is why collaboration is crucial. Only by bringing together the strength of patients and families, researchers, physicians, companies, and other organisations around the world can we accelerate the necessary steps and overcome barriers. Our foundation exists to create those connections.

Our story ANBI – Algemeen Nut Beogende Instelling
ANBI Recognised
Donations are tax deductible in NL
Vision

Together, we want to build a future with more understanding, better care and real hope for everyone living with Limb-Girdle muscular dystrophy.

Mission

Paving the way from care to cure. We work to raise awareness, support patients and families, and accelerate scientific research, so that effective treatments and ultimately a cure for Limb-Girdle muscular dystrophy become possible. We connect people, share knowledge and invest in progress, at a time when clinical trials and innovative therapies such as gene therapy and new medication offer growing hope.

Read more →
Their Stories

Two young men. One shared hope

Tobias and Floris had never met until their fathers were introduced through a mother whose son participated in a clinical trial ten years ago. That story showed that what once seemed impossible could become reality, and ultimately led to the creation of the Together for Limb-Girdle Foundation.

Now, Tobias and Floris share their stories because they believe that by putting a face to the disease, they can help accelerate the journey from hope to treatment for everyone living with Limb-Girdle Muscular Dystrophy.

Tobias
Tobias
Fighting LGMD2A/R1
Read his story →
Floris
Floris
Fighting LGMD2A/R1
Read his story →
The people behind the mission

Meet the parents

Four parents united by the same fight, accelerating the path to treatment for their sons and everyone living with LGMD2A/R1.

Harrie Steenbakkers
Harrie Steenbakkers
Father of Tobias
Emmy Steenbakkers
Emmy Steenbakkers
Mother of Tobias
Berith Behrens Selling
Berith Behrens - Selling
Mother of Floris
Jan Willem Behrens
Jan-Willem Behrens
Father of Floris
Understanding the disease

What is LGMD Type 2A?

Calpainopathy, also known as LGMD Type 2A or LGMD-R1, is a rare genetic muscle disease caused by mutations in the CAPN3 gene. It is the most frequent form of Limb-Girdle Muscular Dystrophy worldwide, affecting more than 100,000 people globally and an estimated 13,000 patients in the US and EU, yet it remains without any approved treatment or cure.

The CAPN3 gene encodes calpain-3, a protein critical for muscle fiber maintenance and repair. When this protein is absent or dysfunctional, muscles progressively deteriorate, beginning in the hips and shoulders. Most patients lose the ability to walk 10 to 25 years after disease onset.

The gene at the root of the disease
CAPN3
Chromosome 15q15.1 · Encodes calpain-3 · Autosomal recessive inheritance

Genetic origin

Caused by mutations in the CAPN3 gene (chromosome 15q15.1). Inherited in an autosomal recessive pattern, meaning both copies of the gene must carry a mutation for the disease to manifest.

Early onset

Symptoms typically appear between ages 8 and 15, in at least two thirds of patients. Onset can occur as early as age 2 or as late as 40, with significant variability even within the same family.

Progressive deterioration

Without treatment, patients lose the ability to walk 10 to 25 years after onset. Physical therapy can slow decline but cannot stop or reverse the underlying muscle loss.

Global impact

More than 100,000 people worldwide live with LGMD-R1/2A, including 13,000 patients in the US and EU. It is the most frequent form of LGMD, and the reason we are focusing our efforts here first.

Living with progression

The race against progression

In LGMD, muscle function can gradually decline over time. People may lose the ability to do the things that matter most.

Young person playing football outdoors

Sports and play

Keeping up with friends and doing the sports you love.

Young person running outdoors

Running

Being able to run, stay active and feel free.

Young person climbing stairs using a handrail

Climbing stairs

Moving around independently at home, school or work.

Young person bending down to pick up an object

Bending over

Simple tasks like picking something up become more difficult.

Young person getting up from a chair

Getting up from a chair

Maintaining independence in daily life.

Young person walking outdoors

Walking

Over time, walking can become very challenging.

While the disease progresses, science is moving forward.

Gene therapy offers real hope to change this trajectory. With the right support, we can help bring treatments within reach faster, for a brighter tomorrow.

Gene therapy research
100K+
Patients worldwide
13,000
In US & EU
CAPN3
Gene mutated
€10M
Next funding goal
Why now

The window is open

For decades, Calpainopathy had no realistic path to treatment. That has changed, thanks to more than a decade of work by Genethon and its spin-off Atamyo Therapeutics.

Genethon is a non-profit pioneer laboratory in the discovery and development of gene therapies for rare diseases. Fifteen gene therapy products developed, alone or with its contribution, are currently in clinical trials across a range of rare diseases. Genethon is also among the originators of Zolgensma®, the gene therapy for spinal muscular atrophy, now licensed to and marketed by Novartis. Their track record is why we believe ATA-300 can make the same journey, from the laboratory to the patients who need it.

"Time is muscle. Every day without a treatment means irreversible muscle loss for thousands of patients worldwide. The science is ready. What we need now is the support to make it happen."
Hope Starts With Action
Current research

The Road to Treatment

Our First Priority

Help bring ATA-300 into a first-in-human clinical trial

10+ years research→ Preclinical development ✓→ Clinical manufacturing→ Phase 1→ 6 first patients
Funding needed: approx. €10 million
Learn More
ATA-300 · LGMD-R1/2A
StatusPreclinical development completed
Next milestoneFirst-in-human Phase 1 clinical trial
Developed byGenethon (Atamyo Therapeutics)
ATA-100: Gene therapy for LGMD-R9/2i (Phase 1b/2b, NCT05224505)
OngoingAtamyo Therapeutics (Genethon)
Learn More
ATA-200: Gene therapy for LGMD-R5/2C (Phase 1b, NCT05973630)
OngoingAtamyo Therapeutics (Genethon)
Learn More
A broader track record of AAV-based gene therapies in clinical development
Track recordGenethon · Fifteen gene therapy products in clinical trials
Learn More
Beyond donating

There are many ways to make a difference

Whether you're a patient, family member, healthcare professional, researcher, or simply someone who believes in rare disease advocacy. There is a role for you in this mission.

Donate

Every contribution goes directly to fund concrete clinical trials that have the potential to make a real impact in a LGMD treatment, and research grants.

Give now

Partner With Us

Are you a research institution, biotech company, hospital, or nonprofit? We actively seek research partnerships and institutional collaborations around the world.

Contact us

Fundraise

Run a marathon, organize an event, or launch a personal campaign. Peer-to-peer fundraising is one of the most powerful ways to spread both money and awareness simultaneously.

Start a campaign
Our network

Connected Organisations

We are proud to be connected with leading patient organisations, research foundations, and advocacy groups around the world working to advance the fight against LGMD.

Scientific connections
Genethon
Atamyo Therapeutics
AFM-Téléthon