September 30: International LGMD Awareness Day
The 10th International Limb-Girdle Muscular Dystrophy (LGMD) Awareness Day takes place on September 30. Organized by the LGMD Awareness Foundation, the day's mission is to draw attention to this group of rare neuromuscular diseases and their impact on the lives of affected children and adults in all regions of the world.
What are Limb Girdle Muscular Dystrophies (LGMD)?
Limb Girdle Muscular Dystrophy (LGMD) is a group of over 30 rare neuromuscular disorders of genetic origin, primarily affecting the muscles of the shoulders (shoulder girdle) and hips (pelvic girdle). Muscles become progressively weaker, leading to difficulties in movement, and some forms of the disease are sometimes accompanied by cardiac or respiratory complications. LGMD is hereditary and can appear at various ages.
At this point in time, treatment is symptomatic, but research into innovative treatments is very active, notably in France through the Telethon laboratories.
Towards treatments for limb-girdle muscular dystrophy
Gene therapy, natural history studies and patient registries... clinical research into limb-girdle muscular dystrophy (LGMD) currently includes seven projects underway or in preparation in France. These include the ATA-100 trial, conducted by Atamyo Therapeutics, a biotechnology company set up by Genethon to accelerate the development of gene therapies for limb-girdle muscular dystrophy, based on research carried out for over thirty years by Isabelle Richard's team at Genethon. The aim of this trial is to evaluate the efficacy and tolerability of a gene therapy treatment in patients aged 16 and over suffering from LGMD R9 (FKRP).
Discover the struggle of the researcher, an expert in limb-girdle muscular dystrophy. (french version)
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The LGMD Interest Group: Support for Patients and Families
Because those directly affected by a disease know it best, the AFM-Téléthon's LGMD interest group brings together patients and families affected by limb-girdle muscular dystrophy. Led by a team of volunteers, the team supports patients and their loved ones, encourages mutual aid and promotes the sharing of experience.
The interest group also provides information on scientific and medical advances, identified by its members at conferences and through exchanges with doctors, researchers and other associations in France and abroad. Thanks to its unique expertise, it contributes to the emergence of research programs and to improving the quality of life of patients and their caregivers.
“World LGMD Awareness Day is essential to put the spotlight on our progressive diseases, which are so rare and disabling on a daily basis, and on the fact that there are no treatments available. By raising awareness and advocating for the needs of people with limb-girdle muscular dystrophies, we hope to improve diagnosis, medical management and the chance of access to future treatments one day.”
Mélanie Bordes, head of the LGMD Interest Group