New Delhi, Nov 4 (NationPress) Motivated by empathy and a dedication to save the lives of children afflicted by a rare and life-threatening genetic condition, Chandni Chowk MP Praveen Khandelwal reached out to Prime Minister Narendra Modi on Tuesday, calling for immediate policy intervention and special authorization to facilitate treatment for LAMA2-related congenital muscular dystrophy (LAMA2-CMD).

LAMA2-CMD is recognized as one of the most uncommon and severe genetic disorders globally, primarily impacting infants and young children. The disorder progressively deteriorates muscle function, rendering many patients incapable of walking, moving, or even breathing independently. Currently, no known cure exists for this condition anywhere in the world.

Khandelwal's motivation is deeply personal; a 20-month-old girl from his constituency is suffering from this ailment. The family, showcasing incredible strength, has discovered approximately 60 other children throughout India who are also battling this disease. All of these children are in desperate need of medical intervention and hope.

In his correspondence, the BJP MP noted that India is at a pivotal moment in medical innovation, akin to the COVID-19 pandemic when the country emerged as a global frontrunner by developing indigenous vaccines.

He emphasized that under Prime Minister Modi's leadership, India can again be a beacon of innovation and empathy by spearheading treatment for LAMA2-CMD.

Referring to recent scientific advancements, Khandelwal highlighted a breakthrough by the Japanese biotech company 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, paving the way for India to potentially be the first nation to initiate human clinical trials for this therapy.

To expedite this possibility, Khandelwal urged the Prime Minister to implement several measures, including: Granting rapid approval for first-in-human clinical trials in accordance with the New Drugs and Clinical Trials Rules, 2019.

He requested the Prime Minister to direct the DCGI and ICMR to collaborate and accelerate the evaluation process.

Furthermore, he suggested recognizing LAMA2-CMD as a distinct category under the National Policy for Rare Diseases, 2021, increasing financial support beyond the existing Rs 50 lakh limit, and fostering partnerships between Indian and international biotechnology firms to hasten gene therapy research.

Khandelwal asserted that decisive government action could provide new hope to affected families and position India as a global leader in next-generation genetic therapies.

He concluded his appeal by expressing, "Your (PM Modi's) compassionate vision has consistently prioritized the welfare of every citizen, particularly the most vulnerable, at the core of India's advancement. We genuinely believe that with your support, India can lead the way in offering hope, healing, and innovation to numerous families grappling with rare diseases."

He also remarked, "Just as India provided hope during the most challenging days of COVID-19, we can now give hope to children born with this rare disorder."