Crowdfunding helps seven-month-old SMA patient access ₹10 crore gene therapy
A seven-month-old infant with Spinal Muscular Atrophy received a ₹10 crore gene therapy treatment at a Bengaluru hospital. The high cost of the life-saving medication was covered entirely through public crowdfunding.
Why it matters
It underscores the extreme financial burden of rare disease treatments and the critical role of public support in bridging healthcare gaps.
A seven-month-old infant diagnosed with Spinal Muscular Atrophy (SMA) at five weeks of age has received a one-time gene replacement therapy at Aster CMI Hospital in Bengaluru through a crowdfunding initiative.
SMA is a rare inherited neuromuscular disorder that progressively affects motor neurons — the nerve cells responsible for controlling muscle movement. It is most commonly caused by abnormalities in the SMN1 gene, resulting in inadequate production of the Survival Motor Neuron (SMN) protein.
Affected infants can develop muscle weakness and have difficulty achieving milestones such as holding their head up, sitting, and moving independently. In severe cases, the muscles involved in swallowing and breathing can also be affected.
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