The drug Zolgensma from the Swiss pharmaceutical company is considered the most expensive drug in the world and is used to treat spinal muscular atrophy (SMA). The disease of certain nerve cells in the spinal cord leads to muscle breakdown. The drug must be given to the little patients before they are two years old. According to the TV channel “Al Arabiya”, the girl from the Egyptian city of Alexandria is one year and eleven months old. Among others, prominent Egyptian businessmen, actors and athletes took part in the collection campaign. Students from the economics faculty at Cairo University also decided not to attend their graduation ceremony and donated the money earmarked for it.
In Austria, newborn screening was expanded to include spinal muscular atrophy (SMA) last year. The genetic defect responsible for the rare disease was discovered in 13 babies. All affected children received treatment within the first three weeks of life.
Source: Nachrichten
