Hope starts with action

The road to a cure

The science is ready. Now hope must become action. Scientific breakthroughs do not change lives on their own. Too often, promising therapies become stuck between successful research and the funding required to enter clinical testing. We exist to bridge that gap.

Hope in action The Phase 1 trial Gene therapy

Team Tobias and Team Floris turning hope into action

We don't raise money for "someday"

We help bring the path to a Phase 1 clinical trial closer. For families living with LGMD, hope cannot remain an abstract concept. Hope must become movement. Progress. Action.

Research is essential. Scientists, doctors and biotech companies are working every day to better understand LGMD and to develop therapies that can change lives. But between a scientific discovery and an actual treatment lies one of the biggest obstacles in the field of rare diseases: funding the path to a clinical trial.

The difference between supporting research and accelerating a clinical trial

Many organisations raise funds for general research. This work is hugely important. It expands scientific knowledge, encourages collaboration and helps to develop new ideas. But research alone does not automatically lead to a treatment for patients.

These steps are costly, time-consuming and often underfunded, particularly for rare diseases such as LGMD. It is precisely here that progress is often delayed. Not because the science lacks potential. But because the path to patients is difficult to finance.

The funding gap

From promising science to patients

Gene therapy has the potential to change lives. But there is a long road from a scientific breakthrough to an approved treatment.

Research
Understanding the disease and developing a therapy.
Therapeutic candidate
A working gene and delivery system.
Manufacturing
Producing the therapy at the required quality and scale.
Clinical trials
Testing safety and effectiveness in people, in phases.
Regulatory pathway
Review and approval by health authorities.
Patients
A safe and effective treatment available to people with LGMD.

Where we are today

Promising science today

Real progress is being made in gene therapy for LGMD.

Clinical trials The essential bridge to a treatment

Funding gap

More support is needed to take promising therapies through clinical trials to patients.

What becomes possible

A brighter tomorrow

A treatment can bring more independence and brighter futures for people with LGMD.

Help close the gap

Turning hope into progress

At the Together for Limb-Girdle Foundation, we want to help bridge that gap. Our mission is not just to raise awareness. Our mission is to help accelerate the path to a Phase 1 clinical trial. That means helping to fund the crucial steps needed to take a promising therapy from the laboratory to the first studies in humans.

Every contribution helps build momentum. Every partnership helps reduce delays. Every supporter helps bring the LGMD community one step closer to real clinical progress.

Accelerating progress: from fundraising to hope and future

Before a therapy reaches a patient

The steps between discovery and a clinical trial

Before a therapy can be tested in humans, many additional steps are required. This is the work that often goes unfunded, and the work our foundation exists to support.

Preclinical validation

Safety and toxicity studies

Production preparation

Regulatory documentation

Trial design and preparation

Collaboration between researchers, doctors and regulators

Why it matters

Why a Phase 1 trial is so important

A Phase 1 clinical trial signifies something fundamental: a potential therapy has been developed to the point where it can be tested in humans for the first time. For rare disease communities, reaching Phase 1 is more than just a scientific milestone.

Proves progress is possible. A potential therapy has been developed to the point where it can be tested in humans for the first time.

Creates visibility. Reaching Phase 1 is more than a scientific milestone. It puts LGMD on the map for partners and investors.

Attracts new investment. A successful Phase 1 makes it easier for additional partners and investors to step in and fund subsequent phases.

Strengthens collaboration. It unites researchers, clinicians, regulators and patient organisations around a shared, concrete goal.

Gives families something tangible. Not a distant hope. But concrete progress.

Time matters

For people living with LGMD, time is not a theoretical concept

The disease progresses every day. That is why accelerating development is so important. Not by compromising on science. But by removing financial barriers that delay promising therapies. The sooner crucial development steps are funded, the sooner the path to a clinical trial can be taken.

"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."

For patients with neuromuscular diseases, the conversation is gradually shifting from "Can gene therapy work?" to "How quickly can we bring effective therapies to patients?" This transition, from scientific possibility to clinical reality, is one of the most significant developments in modern medicine, and offers genuine hope for families affected by rare neuromuscular disorders.

And for families living with LGMD, progress is not measured in published papers. It is measured in steps taken, stairs climbed, independence maintained, and futures regained.

Together we can advance science

No single foundation can solve LGMD on its own. But together, patients, families, researchers, doctors, donors and partners can make a difference.

We believe that hope only truly gains strength when it is linked to action.

And that action can help bring therapies closer to the people who are waiting for them.

Join us

The Together for Limb-Girdle Foundation exists to accelerate progress towards real clinical impact. Because families affected by LGMD deserve more than just awareness.

They deserve momentum

They deserve urgency

They deserve hope backed by action

Together, we can help bring the next step closer.

Making the next step possible

The science is there. The next challenge is getting this progress to patients.

To make the clinical programme for LGMD R1/2A possible, an estimated €10 million is needed. That is not an amount that a single foundation, organisation or donor should have to raise alone. It takes collaboration between patient organisations, funds, donors and other partners.

Together for Limb-Girdle wants to help bring those forces together and so help speed up the road to clinical trials.

Every contribution helps make the next step possible.

Be part of the next step

Your donation, large or small and from anywhere in the world, helps fund the path to a Phase 1 clinical trial for LGMD2A/R1, the first systemic gene replacement approach designed specifically for people with Calpainopathy.

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