Bengaluru Infant Receives Life-Saving Gene Therapy for SMA

A seven-month-old infant in Bengaluru has successfully received a one-time gene replacement therapy for Spinal Muscular Atrophy (SMA). The complex treatment, costing approximately ₹10 crore, was entirely funded through public contributions. This medical milestone highlights the potential of advanced therapies and the generosity of community support.

SMA, a rare inherited disorder, affects motor neurons essential for muscle movement. Early diagnosis is crucial, as demonstrated when this infant was identified with the condition at just five weeks old. This allowed for prompt initiation of disease-modifying treatments.

The infant was initially managed with risdiplam and supportive care. Subsequently, the gene therapy, onasemnogene abeparvovec, was administered. This therapy works by delivering a functional copy of the SMN1 gene to help produce the necessary protein for motor-neuron survival.

The successful crowdfunding effort enabled the family to access this vital treatment, which is often prohibitively expensive. This case underscores the critical role of early detection and timely intervention in improving outcomes for children with SMA.