Delivering treatments for Spinal Muscular Atrophy in Latin America
His name is Santiago, her name is Amelia1. While they live 3,000 kilometres apart, they both share a similar story. By the time they were six months old, they were diagnosed with spinal muscular atrophy (SMA), a rare genetic disease found in 1 in 10,000 children born worldwide and which, if left untreated, may lead to death or the need for permanent ventilation by the age of two, in more than 90% of cases2.
Like many other families facing this devastating news had done before them, Santiago and Amelia’s parents, living respectively in Brazil and Chile, entered a race against time in order to raise enough money to obtain a one-time, potentially life-saving treatment before their child would turn two years old. This treatment, a gene replacement therapy produced in the US, might have the capacity to repair the faulty genes and extend both children’s life expectancy.
No room for error
Such therapies often require complex logistics to deliver, contending with limitations such as 48-hour shelf lives, or precise temperature ranges, in this case -70ºc. Any excursion can not only cost millions of dollars, but above all have catastrophic consequences for the patients.
As the company responsible for transporting this medicine to Santiago and Amelia1, we were honored to see our teams in Brazil and Chile go above and beyond to ensure safe and timely deliveries to the patients.
wanted to make sure that nothing would go wrong so we also asked for a police
escort from the airport to the hospital to ensure that we would be there on
Jaqueline Escotero, Regional Vice President, Latin America
1 Names have been modified to protect the identities of the children portrayed in the story.
2 Finkel RS, et al. Neurology. 2014;83(9):810-817.