According to YAHOO News, twenty-two-month-old Devdan has a rare disease called type 2 spinal muscular atrophy (SMA2) which affects nerve cells that control muscles. If left untreated progressive muscle weakness develops. SMA2 patients are unable to walk or stand without assistance and have problems speaking, breathing, and swallowing.
If Devdan were unable to receive the drug the only other options for him would be daily oral medication and injection into the spinal canal every four months for life.
Zolgensma, a one-time therapy, is the most expensive drug in the world with a price tag of $2.868 million. It was developed for children under the age of two. Early data looks promising. Zolgensma has been approved by the FDA but has not yet been approved by the Health Sciences Authority of Singapore. Therefore, the drug must be imported under a Special Access Route in order to be administered in Singapore.
Zolgensma targets the fundamental cause of SMA2 by identifying and restoring the root cause of the disease. The patient receives a one-time infusion into the spinal canal which brings about a response in motor function. Infants and toddlers who have been given Zolgensma have gained the ability to walk and stand independently.
Shu Wen and Dave Devaraj, Devdan’s parents, can now pay for Zolgensma therapy thanks to the generosity of the many donors who responded to their public appeal on August 3rd. The sum of $2.868 million was raised in ten days by over 29,000 people through the Ray of Hope website for charities. Dave and Shu, both age 33, say that they are so grateful for the love, support, and generosity shown to their little boy. They say that Devdan’s life was saved.
Devdan will receive his treatment shortly at the National University Hospital.