The 30th of September is Limb-Girdle Muscular Dystrophy Awareness Day. At this occasion, the LGMD Interest Group of the AFM-Téléthon is planning an information day about these diseases in Evry.
Limb-Girdle Muscular Dystrophy (LGMD) Awareness Day is intended to be an annual collaborative effort to globally raise awareness of individuals living with limb-girdle muscular dystrophy and to get the attention of researchers, health care providers, or the general public, that it deserves.
"Together we are stronger" is the theme of the day.
On the same day, the LGMD Interest Group of the AFM-Téléthon is planning an information day about limb-girdle muscular dystrophy at the Génocentre in Evry. An informative plenary session, workshops, meetings with clinicians and researchers, exchanges between professionals... no doubt the day will be rich and informative!
Limb-girdle muscular dystrophies: 32 different diseases
So far, 32 types of limb-girdle muscular dystrophy (LGMD) have been identified. These are rare diseases that mainly affect the muscles of the shoulders and hips and can progress to the point where a person loses the ability to walk with sometimes cardiac or respiratory complications.
It is estimated that between 1 and 3 out of every 125,000 people are affected by the disease.
Thanks to the work carried out for over 30 years by Isabelle Richard, a researcher specializing in limb-girdle muscular dystrophy at Genethon, the AFM-Telethon's dedicated gene therapy laboratory, the era of gene therapy is opening up for this group of genetic muscular diseases. After increasing the number of proofs of concept, the researcher and her team have demonstrated the efficacy of gene therapy in preclinical stages, for limb-girdle muscular dystrophy linked to the FKRP gene.
The first European trial for limb-girdle muscular dystrophy linked to the FKRP gene has been launched. And several other innovative therapy trials are currently underway for these diseases around the world!