New Delhi, Nov 4 Driven by compassion and a mission to save the lives of children battling a rare and fatal genetic disorder, Chandni Chowk MP Praveen Khandelwal, on Tuesday, wrote to Prime Minister Narendra Modi seeking urgent policy intervention and special approval to enable treatment for LAMA2-related congenital muscular dystrophy (LAMA2-CMD).
LAMA2-CMD is among the world's rarest and most devastating genetic disorders, primarily affecting infants and young children. The condition progressively weakens muscles, leaving many patients unable to walk, move, or even breathe without assistance. Currently, there is no known cure for the disorder anywhere in the world.
Khandelwal's appeal stems from a personal encounter, a 20-month-old girl from his constituency is suffering from this disease. The child's family, demonstrating remarkable courage, has identified around 60 other children across India afflicted with the same disorder. All of them are in urgent need of medical hope and intervention.
In his letter, the BJP MP stated that India stands at a historic crossroads in medical innovation, similar to the COVID-19 pandemic, when the nation emerged as a global leader by developing indigenous vaccines.
He said that under Prime Minister Modi's leadership, India could again become a symbol of innovation and compassion by pioneering treatment for LAMA2-CMD.
Highlighting recent scientific progress, Khandelwal referred to a breakthrough by Japanese biotech firm Modalis Therapeutics, which has developed a promising CRISPR-based gene-editing therapy for the disorder. The Indian Council of Medical Research (ICMR) has already classified this technology as low-risk, opening the door for India to become the first country to begin human clinical trials for the therapy.
To fast-track this possibility, Khandelwal urged the Prime Minister to take several steps, including: Granting expedited approval for first-in-human clinical trials under the New Drugs and Clinical Trials Rules, 2019.
He asked the Prime Minister to direct DCGI and ICMR to collaborate and fast-track the evaluation process.
Recognising LAMA2-CMD as a distinct category under the National Policy for Rare Diseases, 2021, and increasing financial aid beyond the current Rs 50 lakh cap and promoting collaboration between Indian and international biotechnology institutions to accelerate gene therapy research.
Khandelwal said that with decisive government action, India could bring new hope to affected families and position itself as a global leader in next-generation genetic therapies.
He concluded his appeal by saying, "Your (PM Modi's) compassionate vision has always placed the well-being of every citizen, especially the most vulnerable, at the heart of India's progress. We sincerely believe that with your support, India can lead the way in bringing hope, healing, and innovation to countless families battling rare diseases."
He also mentioned that "Just as India gave the world hope during the darkest days of COVID-19, we can now give hope to children born with this rare disorder".
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