Spinal Muscular Atrophy (SMA) Type 1 is a rare genetic disease. It is caused by loss of nerve cells, which carry electrical signals from the brain to the muscles.
- The protein needed for this signalling is coded by a gene for which everyone has two copies – one from the mother and the other from the father.
- A child develops this disorder only if both the copies are faulty. Without treatment, this disease is ultimately fatal.
- The therapy for this rare disease is Zolgensma which is a one-time infusion that takes about an hour.
- The therapy for SMA was approved by U.S. regulators in May 2019 and has since then turned into a miracle drug for this rare disorder that destroys a baby’s muscle control.
(Source: The Hindu)
