In New Delhi, Resident Councillor Praveen Khandelwal, the MP for Chandni Chowk, sent a letter to Prime Minister Narendra Modi asking for urgent help to treat children with a deadly rare disorder called LAMA2‑related congenital muscular dystrophy (LAMA2‑CMD).
LAMA2‑CMD is one of the world’s rarest genetic diseases. It starts in babies and early‑childhood, slowly eroding muscle strength until many sufferers can’t walk, move, or even breathe without support. No cure exists yet, and families desperately need new options.
Khandelwal’s plea comes from a personal story: a 20‑month‑old girl in his constituency is being diagnosed with LAMA2‑CMD. She isn’t alone; researchers have found about 60 other Indian children with the same disease.
The MP urges India to follow the example set during COVID‑19, when the nation built a leading vaccine program from scratch. He says the country has the chance to be a world leader again—this time in gene therapy for LAMA2‑CMD.
He points to a breakthrough by Japan’s Modalis Therapeutics, which has created a CRISPR‑based gene‑editing therapy for the disorder. The Indian Council of Medical Research (ICMR) already marked this technology as low‑risk, clearing the way for India to start human trials first in the world.
Khandelwal wants the government to act quickly. He asks the Prime Minister to:
* Fast‑track approval for the first human clinical trials under the 2019 New Drugs and Clinical Trials Rules.
* Direct the Drugs Controller General (DCGI) and ICMR to speed up review.
* Recognise LAMA2‑CMD as a separate category in the 2021 National Policy for Rare Diseases.
* Increase the current ₹50 lakh funding cap.
* Encourage partnerships between Indian and international biotechs.
“If decisive action is taken, India can give new hope to families and become a global leader in next‑generation genetic therapies,” Khandelwal wrote.
He concluded by reminding the Prime Minister that India’s compassionate vision has always put the most vulnerable first. He urged Modi to help usher in a new era of hope for children with rare diseases, just as India did during the darkest days of the COVID‑19 pandemic.
Source: ianslive
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