WHAT LGMD SUB-TYPE DOES YOUR ORGANIZATION FOCUSES ON:
We focus exclusively on LGMD2I
IF YOU FOCUS ON A SUB-TYPE, IS THERE A REGISTRY AND HOW CAN PATIENTS JOIN?
We are aware of one registry: the Global FKRP Registry (https://www.fkrp-registry.org/). While it is based in the UK, it is an international registry, adapted to the nature of rare diseases. LGMD2I is a very rare disease, which means that the number of people affected by it is small from the point of view of drug developers. Because advanced clinical studies require a lot of participants, having a global registry will help identify a significant number of potential participants and increase the chance of successful enrollment in much-needed trials. We have financially supported this registry for many years and it is becoming a great asset for the LGMD2I community.
Is your organization a Non-Profit? If so, what type:
Yes, we are a non-profit private foundation.
WHAT INSPIRED THE CREATION OF YOUR ORGANIZATION:
People with LGMD2I have been influential in defining our focus and mission.
wHAT IS YOUR ORGANIZATION’S MISSION:
To find a cure and help people with 2I maintain their quality of life.
WHAT SERVICES DOES YOUR ORGANIZATION PROVIDE:
We do not provide services. We fund research and help connect people to information about the disease and medical care.
WHAT IS YOUR ORGANIZATION MOST PROUD OF:
We have funded the initiation of programs in all of the main therapy fields (gene therapy, stem cells, …), diagnostics, and research tools (mouse model, registry, …). Thanks to our involvement, the number of researchers in LGMD2I has grown steadily in all regions of the world. It was important to create a certain critical mass of research groups; now it is important to keep it going…
What do you want the world to know about YOUR ORGANIZATION:
We fund any LGMD2I research projects that can bring us closer to a cure, and we support large-scale diagnosis. We believe that the appropriate treatment/cure for 2I will be a combination of modalities that will fix the faulty gene and help revert the damage made to the muscles. Any laboratory that has research know-how and technologies that can be applied to developing a treatment for LGMD2I should contact us.
HOW CAN PEOPLE BECOME INVOLVED IN SUPPORTING YOUR ORGANIZATION:
Anyone with information about the disease or who is interested in finding a cure is welcome to get in touch with us.
WHAT IS THE BEST WAY TO CONTACT YOUR ORGANIZATION:
For more information, please contact: Dr. Jean-Pierre Laurent, Program Officer via email: firstname.lastname@example.org
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