Mumbai: Meet Khushi Kumari Yadav, a 20-month-old little warrior diagnosed with Spinal Muscular Atrophy (SMA) Type 2. Despite being a cheerful and curious toddler, Khushi faces daily struggles due to severe muscle weakness, making even simple movements challenging. While she can sit with support, she cannot stand or walk and requires constant care and specialized medical attention.
To halt the progression of her disease, Khushi urgently needs a life-saving gene therapy, Zolgensma, which costs ₹16 crore, with an upfront payment of ₹9.5 crore an amount far beyond her family’s means.
Understanding SMA
SMA is a rare genetic disorder caused by the deletion of the SMN1 gene, crucial for the survival of motor neurons. Without this gene, these neurons gradually die, leading to progressive loss of muscle control starting from the legs and waist, then the hands, and eventually affecting the spine and respiratory system.
While the brain remains unaffected, the disease severely limits mobility and independence.
A medical breakthrough brings new hope
Until a few years ago, SMA had no cure, and parents were often told to take their children home and make the most of their limited time together. However, medical science has since made a revolutionary breakthrough Novartis Pharmaceuticals developed Zolgensma, a one-time gene therapy that can potentially halt SMA’s progression and give children like Khushi a chance at a normal life.
An official letter from the Consultant Pediatric Neurologist at P.D. Hinduja Hospital, Mumbai, confirms that Khushi requires this life-saving injection, which is not available in India and must be imported from the USA.
A race against time
Khushi’s condition is rapidly worsening, affecting her ability to sit, crawl, and breathe independently. Early intervention is critical, as Zolgensma must be administered before the age of two.
Her father, Samit Yadav, shared that the total cost of Zolgensma is ₹16 crore, with ₹9.5 crore required upfront to Novartis and the rest payable in instalments. To make this treatment possible, Khushi’s family has turned to the public through crowdfunding, reaching out to individuals, organisations, and well-wishers.
An emotional appeal for help
“The medicine must be administered before Khushi turns 24 months to achieve the best results. Being 20 months old, she has only four months left,” said Samit.
“We are doing everything possible to save Khushi’s life,” he added. “Every contribution, big or small, brings her one step closer to strength and mobility.”
This is an urgent appeal to humanity to come forward and support Khushi in her fight for survival. With timely help, this little girl can overcome the limitations of SMA and enjoy a healthier, more active childhood.
