Cairo — Roqaia Reda was born on July 13, 2020, in Alexandria, Egypt. She seemed a happy and healthy baby girl for her first six months, but then her parents started noticing some unusual symptoms. After a series of tests, Roqaia was diagnosed with spinal muscular atrophy (SMA), a rare degenerative neuromuscular disorder that leaves patients without control of their bodies.
In the most severe cases, which are generally those that manifest in early childhood, patients often die before they reach their second birthday. Most people with moderate cases live into adulthood, but they're often left unable to walk.
There are a couple of drugs to treat SMA in young children, the most effective being a single-dose, intravenous treatment of Zolgensma. The gene therapy, which was only approved for use on young SMA patients in the United States in 2019, is often referred to as the most expensive drug in the world. Manufacturer Novartis puts the price tag for treatment at about $2.1 million.
While finding that kind of money would be difficult for most families around the world, there is also little time to do so, as it must be administered to the child before they turn 2.
For Roqaia's parents, it was impossible to fathom, especially as her second birthday was just a couple of weeks away. For perspective, the minimum wage in Egypt is about $145 per month. A family with two parents on that kind of wage would have to save for more than 600 years to afford the treatment.