A Global Call for Hope: 20-Month-Old Khushi Needs Life-Saving Gene Therapy Before It’s Too Late. As the clock ticks down, a family appeals to the world to save their only child from a rare genetic disease that can be stopped—if help arrives in time.
Mumbai, India / November 2025 — Twenty-month-old Khushi Kumari Yadav should be chasing her first steps and giggling at her parents’ voices. Instead, she is fighting for her life against Spinal Muscular Atrophy (SMA) Type 2, a rare genetic disorder that slowly weakens every muscle in her body — taking away her ability to move, eat, and eventually breathe. The only treatment that can stop this disease is Zolgensma, a one-time gene therapy known as the most expensive drug in the world. It costs about $2.1 million and must be administered before a child turns two years old. Khushi is now 20 months old. The clock is ticking. “This is bigger than what one family can carry,” says Manee Kamboj, Khushi’s aunt and a global strategy executive based in the United States. “We’re asking the world not just for money, but for compassion — to share her story and stand with her in this race against time.” Khushi’s father, Samit Yadav, has faced many challenges, but nothing compares to watching his only child lose her strength day by day. “She is our world,” he says quietly. “We just want her to have a chance at life.” SMA is caused by a missing or defective SMN1 gene, which prevents the body from producing a protein critical for the survival of motor neurons — the cells that control muscle movement. Without it, those neurons die, leading to progressive muscle wasting and paralysis. In advanced stages, the muscles that control swallowing and breathing fail, leading to respiratory complications, organ collapse, and ultimately death.
Zolgensma works by delivering a functional copy of the SMN1 gene to restore that essential protein, effectively halting the disease. When given early, it can allow children to walk, play, and live full lives — milestones once thought impossible. “This therapy is science’s miracle, but it comes with a cost beyond reach,” Kamboj says. “Still, we’ve seen what happens when humanity unites. Every share, every act of kindness brings her closer to the miracle she needs.” Khushi’s family has launched verified fundraising campaigns — one for supporters in the United States and abroad, and one for those in India. They’re appealing to individuals, corporations, and foundations to help bridge the gap and give Khushi her only chance at survival. Spinal Muscular Atrophy is one of the leading genetic causes of infant mortality worldwide, affecting roughly one in 10,000 births. It is caused by the absence of the SMN1 gene, leading to progressive muscle weakness, respiratory decline, and, if untreated, fatal organ failure.
How to Help:
🌎 For friends in the U.S. or outside India:
🇮🇳 For friends donating from India:
https://www.impactguru.com/fundraiser/please-help-khushi-yadav
Follow and Share Khushi’s Journey
Instagram: http://@khushi_fights_sma
X (Twitter): http://@Khushi_SMA2
