Limb-girdle muscular dystrophy type 2I/R9
Limb-girdle muscular dystrophy 2I/R9 (LGMD 2I/R9) is a term for a group of inherited diseases that cause weakness and wasting of the muscles in the arms and legs. The muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area and thighs. The severity, age of onset, and features of LGMD 2I/R9 vary among the many subtypes of the condition.
LGMD 2I/R9 is caused by mutations in the Fukutin Related Protein (FKRP) gene. Currently, there is no cure, and treatment is based on the signs and symptoms present in each individual. In LGMD 2I/R9, specifically, signs and symptoms often develop in late childhood and may include difficulty running and walking. The symptoms gradually worsen over time, and affected people generally rely on a wheelchair for decades.
Visit the National Institutes of Health Medline Plus website to learn more about Limb-Girdle Muscular Dystrophy R9.
Limb-Girdle Advocacy Partners
We are fortunate to work with patient advocacy groups, as well as leading researchers, scientists and medical professionals to help bring new therapeutics for LGMDR9 and other forms of muscular dystrophy into the clinic.
Clinical Trials
Many patients with genetic diseases may suffer an altered way of life. Many of these diseases are progressive. Thus, time is of the essence and the importance of every clinical trial has greater meaning to all of us at AskBio.
