Baby Faziq, a two-year-old Singaporean boy, has received a groundbreaking S$2.4 million treatment for a rare genetic disorder known as spinal muscular atrophy (SMA) Type 1. The treatment, Zolgensma, is a gene therapy that is considered one of the most expensive drugs in the world.
Faziq was diagnosed with SMA Type 1 at four months old. The disease affects the motor neurons in the spinal cord, leading to progressive muscle weakness and paralysis. Without treatment, SMA Type 1 is often fatal within the first two years of life. The S$2.4 million cost of Zolgensma was covered through a combination of public donations and contributions from various organizations.
A significant portion of the funds was raised through a crowdfunding campaign that garnered widespread support from the Singaporean community. The gene therapy aims to replace the faulty SMN1 gene responsible for SMA. Administered as a one-time infusion, Zolgensma is designed to halt the progression of the disease and, in some cases, even reverse some of the muscle weakness.
Faziq received the treatment at the Khoo Teck Puat-Hospital. The successful treatment marks a significant milestone in pediatric medicine and offers hope to families affected by rare genetic diseases. The high cost of such advanced therapies, however, continues to be a challenge for healthcare systems worldwide.
