In a bid to save the lives of children suffering from a rare and life-threatening genetic disorder, Member of Parliament from Delhi’s Chandni Chowk, Praveen Khandelwal, has written to Prime Minister Narendra Modi, seeking special policy intervention and fast-tracked approval for the treatment of LAMA2-related Congenital Muscular Dystrophy (LAMA2-CMD).
LAMA2-CMD is among the world’s rarest and most devastating genetic disorders, affecting infants and young children, often depriving them of the ability to walk, move, or even breathe independently.
Khandelwal’s appeal stems from a deeply personal experience involving a 20-month-old girl from his constituency who is currently battling this cruel disorder.
According to the MP, this is not an isolated case — the affected family has so far identified around 60 children across India suffering from the same condition, all awaiting a ray of hope.
In his letter, Khandelwal stated that the country currently stands at a historic juncture to once again lead the world in healthcare innovation — just as it did during the COVID-19 pandemic, when under Prime Minister Modi’s leadership, India not only developed its own vaccines but also emerged as a beacon of hope for humanity.
The MP also highlighted a recent breakthrough by a Japanese biotech firm that has developed a promising CRISPR-based gene-editing therapy for LAMA2-CMD.
He noted that the Indian Council of Medical Research (ICMR) has already classified this technology as low-risk, creating a unique opportunity for India to pioneer its clinical application.
Khandelwal urged the Prime Minister to grant expedited approval for the first-in-human clinical trial under the New Drugs and Clinical Trials Rules, 2019.
He also requested the Prime Minister to direct the Drugs Controller General of India (DCGI) and ICMR to collaborate and fast-track the evaluation and approval process.
Furthermore, Khandelwal appealed for LAMA2-CMD to be recognized as a distinct category under the National Policy for Rare Diseases, 2021, and sought an increase in financial assistance beyond the existing ₹50 lakh cap.
He also called for greater collaboration between Indian and global biotechnology institutions to accelerate gene therapy research and innovation.