A one-year-old boy with a rare disease has died four months after receiving the most expensive drug in the world.

Derek Pinargote passed away on Sunday, 23rd January, with the sad news reported by his parents on social media.

The youngster suffered from spinal muscular atrophy (SMA), and he was one of only 20 children in his home country of Ecuador with the genetic disorder, which is characterised by weakness and wasting in muscles used for movement.

Derek, who was born in the western Ecuadorian province of Manabi, passed away four months after receiving the world’s most expensive drug to treat his condition.

Despite receiving the drug, he still had to be hospitalised several times, including to have a gastrostomy feeding button fitted.

His mother Genesis Pinargote launched a fundraising campaign at the time to buy the gene therapy medication onasemnogene abeparvovec, which is sold under the brand name Zolgensma, for USD 2 million (GBP 1.48 million).

The family’s efforts managed to get the Ministry of Health to give them the medication on 28th October 2021, with Derek celebrating his first birthday shortly afterwards, on 7th November.

The youngster was reported to be increasingly mobile in December and was also reported to have responded well to an operation on 15th January to remove a feeding tube that had been causing him discomfort.

However, he passed away from unknown causes on Sunday.

Genesis wrote on social media: “God, give us strength to accept your wishes. Today our Derek is by your side. I would like to understand why, but only You will give us strength.”

The earlier a person develops SMA, the more severe the symptoms, which include floppy or weak arms and legs, movement problems, twitching or shaking muscles, bone and joint problems, swallowing problems and breathing difficulties.