Written By: Prerna Mittra | Updated : December 18, 2023 6:02 PM IST
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LGMD encompasses a diverse set of genetic muscle disorders, with over 30 identified subtypes, each caused by mutations in different genes. (Photo: Freepik)
Limb-girdle muscular dystrophy (LGMD) is a group of rare genetic disorders that affect the muscles responsible for movement. Patients suffering from LGMD often live a life filled with physical limitations and challenges. According to medlineplus.gov, LGMD causes weakness and wasting of the muscles in the arms and legs. The muscles most affected are those closest to the body (proximal muscles), such as muscles of the shoulders, upper arms, pelvic area, and thighs.
While traditional treatments have focused on managing symptoms with medications, Dr Pradeep Mahajan, regenerative medicine researcher and founder of StemRx BioScience Solutions India -- who is pioneering a "revolutionary approach that offers new hope to LGMD patients" -- says there will soon be cells rather than pills, and patients will be able to carry their own repairing kits in their body.
"LGMD encompasses a diverse set of genetic muscle disorders, with over 30 identified subtypes, each caused by mutations in different genes. These mutations lead to the progressive weakening of muscles in the shoulders, upper arms, pelvis, and thighs, causing mobility issues and decreased quality of life. LGMD patients typically rely on physical therapy, assistive devices, and medications to manage their symptoms," explains Dr Mahajan.
According to the doctor, regenerative medicine offers promising avenues for the treatment of patients with LGMD. "By harnessing cutting-edge techniques such as stem cell therapy and gene editing, regenerative medicine holds the potential to restore or enhance muscle function in LGMD patients."
He adds that stem cells can be used to repair damaged muscle tissues, and that these innovative approaches offer hope for slowing the disease's progression, improving patients' quality of life, and potentially even providing long-term solutions for those affected by this debilitating condition. "Traditional treatments primarily aim at managing symptoms and slowing down the disease's progression. I believe our bodies have an inherent capacity to repair and regenerate damaged tissues. At StemRx, we are working towards harnessing these innate regenerative capabilities to provide long-term solutions for LGMD patients," Dr Mahajan tells this outlet.
The idea, he adds, is to use the patient's own cells as the building blocks for regeneration. Stem cells, in particular, have shown immense promise in regenerating damaged muscle tissue. As mentioned earlier, one of Dr Mahajan's core beliefs is that the human body carries its own repairing kits. "We don't need to rely on external interventions as much as we need to tap into the inherent potential of our bodies. Stem cells and other regenerative treatments act as catalysts, kickstarting the natural repair processes."
The doctor concludes by saying that while further research and clinical trials are needed to solidify the effectiveness of these treatments, they offer a "promising future for those affected by LGMD". They also bring us one step closer to a world where muscular dystrophy is no longer an insurmountable challenge, but a condition that can be effectively managed and even reversed, he states.
