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Indian healthcare industry is confronting paucity of trained neuromuscular specialists in early detection and treatment of Duchenne muscular dystrophy (DMD), a genetic disorder causing progressive muscle degeneration. This has resulted in despair across families in the country where DMD is diagnosed.
From unaffordable treatment cost estimated to be anywhere around Rs. 4 crore per patient, lack of targeted therapy together with shortage of appropriate training on joint contraction management and early detection of cardio myopathy, India is now looking at multidisciplinary camps to diagnose and counsel DMD patient families , said Prasanna Shirol, co-founder and executive director, Organisaiton of Rare Diseases of India (ORDI).
According to Dr Ann Agnes Mathew, neuro muscular consultant, Bangalore Baptist Hospital and partner, Synapse Neuro Center, there are around 51 ongoing clinical trials for DMD globally and many of these have started entering India. No more details can be shared because it is under the non-disclosure agreement ambit.
Pharmabiz gathers that the clinical trials for DMD for which patient enrolment is on will look for newer types of gene therapies or molecular interventions. Some of the human studies would also assess how cognition, muscles and cardiac functions are affected.
Another major development in India is that early this year, the Indian Institute of Technology (IIT), Jodhpur set up a research centre for DMD in collaboration with Dystrophy Annihilation Research Trust (DART), Bengaluru and the All India Institute of Medical Sciences (AIIMS) Jodhpur. The centre aims to develop affordable therapeutics for this rare and incurable genetic disorder as India accounts for over 5 lakh cases.
There is an imminent need to build team of gene therapy experts to handle neuro muscular diseases and disorders. The big challenge in India is that access to the medicines are difficult and exorbitant costs deter the poor patient families to even consider treatment, said Shirol.
Medical experts noted that treatments available for DMD globally are not available in India. Different treatments will be useful at different times as a therapeutic window. When the inflammation sets in more often steroids are administered on these patients.
Even for the lone approved gene therapy available recently, the drugs for this are the costliest in the market and not available in India. Moreover, it needs to be administered before the age of 6 to get the adequate benefit. The doctor has to be trained on what to monitor and understand the abnormalities for kidney functions among others. Therefore, management of DMD which occurs between the ages of 2 and 4 years but manifests later in childhood requires to be managed in a holistic manner. This is where a multidisciplinary camp comprising access to paediatric neurologist, nutritionist, neuromuscular physiotherapist, to provide long-term quality care for the patient is seen a viable solution for now, said the medial experts.
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