We focus our efforts on one of the most critical gaps in the development process: helping promising therapies move from the laboratory into clinical trials where they can begin to change lives.
Many rare disease foundations support a wide range of valuable activities, from patient support and awareness campaigns to basic scientific research. While these efforts are important, they often spread limited resources across many small initiatives, making it difficult to generate the momentum needed to bring new treatments to patients.
At the Together for Limb-Girdle Foundation, we have chosen a different path.
Our mission is to pave the way from care to cure. That means we focus our efforts on one of the most critical gaps in the development process: helping promising therapies move from the laboratory into clinical trials where they can begin to change lives.
Rather than funding research in a fragmented way, we concentrate on carefully selected clinical programs that have the potential to make a meaningful difference for people living with Limb-Girdle Muscular Dystrophy (LGMD). We look for opportunities where additional funding, advocacy and collaboration can accelerate the journey toward human trials and ultimately approved treatments.
Limited resources spread thinly across many valuable but fragmented activities, making real momentum toward treatments hard to achieve.
We concentrate funding on carefully selected clinical programs, accelerating the journey from the laboratory toward human trials and approved treatments.
We believe that every euro donated should have the greatest possible chance of advancing a therapy toward patients. Our approach is guided by three principles:
We prioritize programs that are moving toward or are already in clinical development.
We focus on therapies with the potential to significantly improve quality of life and disease progression.
We support approaches that can generate knowledge, technology and clinical experience applicable beyond a single subtype of LGMD.
The challenges faced by people with LGMD are often shared across many forms of muscular dystrophy. Advances in gene therapy, clinical trial design, manufacturing and regulatory pathways developed for one condition can help accelerate progress for others.
Success in one muscular dystrophy can help unlock opportunities for many others.
Too often, promising therapies become stuck between successful research and the funding required to enter clinical testing.
We focus on bridging that gap.
By bringing together patients, families, researchers, clinicians, industry partners and donors, we help move the most promising therapies closer to the people who need them most. Our goal is not simply to fund research. It is to help deliver treatments.
Because 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.
For families living with LGMD, what matters most is time together, and the hope that the next generation will have more of it. Real progress isn't an abstract milestone in a laboratory. It is a stair climbed, a journey made, an independence kept. That is the change we are working to make possible.
That is how we pave the way from care to cure.
Every contribution helps move the most promising therapies closer to the people who need them most, bridging the gap between the laboratory and the patients who are waiting.