New Delhi, November 4 (IANS). In a bid to bring hope to children suffering from a rare and life-threatening genetic disorder, LAMA 2-related congenital muscle disorder (LAMA 2-CMD), Chandni Chowk MP Praveen Khandelwal has written to Prime Minister Narendra Modi, appealing for special policy intervention and early approval.
LAMA 2-CMD is one of the world’s rarest genetic disorders, affecting newborn babies and destroying their muscle function, leaving them unable to walk, move or even breathe.
MP Praveen Khandelwal said that a 20-month-old girl from his area is suffering from this disease and so far around 60 children have been found suffering from this disorder across the country.
The MP said that India can play the same leadership role in medical innovation today as it did during the Covid-19 vaccine development. He mentioned a new CiSPR-based gene-editing therapy developed by Japanese biotech company Modalis Therapeutics, which has been classified as a low-risk technology by the Indian Council of Medical Research (ICMR).
MP Khandelwal believes that with early government intervention, India can become the first country in the world to start human clinical trials of this treatment.
In his letter, he has asked Prime Minister Modi for early approval of the first human trial under the New Drugs and Clinical Trials Rules, 2019, DCGI and ICMR to speed up the evaluation and approval process, increasing the financial assistance beyond Rs 50 lakh by including LAMA 2-CMD in a separate category in the National Rare Disease Policy, 2021, and encouraging collaboration between Indian and global biotechnology institutions.
Khandelwal said that this step under the leadership of Prime Minister Modi will prove to be a historic milestone in India’s healthcare journey.
Appealing to Prime Minister Modi, he said, “Just as India gave hope to the world in the darkness of Covid-19, in the same way these innocent children can now be given a new ray of life.”
–IANS
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