In two days more than 950,000 Belgium, almost one in 10 of the country's population, people pledged US$2.1 million for the single course of treatment that may save the life of Pia Boehnke that is only available only in the US.

By Ian Horswill

Posted on September 19, 2019

It took just two days as almost one in 10 people in Belgium paid a total of US$2.1 million to try and save the life of baby Pia Boehnke, who was born with a deadly genetic neuromuscular condition.

Pia Boehnke, born on 27 November last year, was diagnosed in May with Type 1 spinal muscular atrophy (SMA), a neuromuscular disorder that affects the nerve cells that control voluntary muscles. It is also known as Werdnig-Hoffmann disease.

Without treatment, Pia Boehnke would be dead by age two. Her parents live in Antwerp, Belgium, and the drug, Zolgensma, is awaiting approval in Belgium. The parents decided their only option was to take Pia to the US and pay the full price for the drug treatment – US$2.1 million.

Pia’s mother Ellen De Meyer noticed something was amiss in Pia’s development. She did not move around much and could not hold up her head.

“SMA. 3 letters that changed our lives,” De Meyer wrote in July on the the website Team Pia, where they launched a fund-raising campaign to raise awareness of her condition and to try and raise the money for their daughter’s treatment.

Pia Boehnke

Pia is being treated with another new drug, Spinraza, which is keeping her alive.

Then “a wondrous medicine was invented, called Zolgensma,” her parents Ellen and Tim Boehnke wrote in her first-person voice. “It’s a gene-therapy, administered in one shot, that will put the SMN-1 gene I’m missing into my DNA. The first results of this medicine are very promising. I could lead a long, beautiful life, in which I might even learn to walk, or at least be more independent.”

The US Food and Drug Administration (FDA) had approved Zolgensma but her treatment was not covered by their insurance. The couple went to the drug manufacturer Novartis, based in Switzerland, and Pia’s parents were told they would have to find US$2 million for the drug that would save Pia’s life.

The treatment is a “one-off, transformative therapy for an extremely rare disease,” Novartis told BBC News. “We are aware of Pia and the efforts to facilitate her access to treatment. We also recognise that patients and families around the world are interested in gaining access to treatment as quickly as possible.”

A clinical trial of Zolgensma is being conducted in Belgium but Pia’s application was two weeks’ too late.

“I’m currently the only child in Belgium not in such a trial, and thus the only one that doesn’t get this medicine,” Team Pia wrote.

Two fundraising methods were organised at the start of the week: a text-messaging donation app and a GoFundMe page.

As of Wednesday, more than 950,000 Belgium people pledged US$2.1 million for the single course of treatment.

“It is overwhelming that a country can come together for something like this,” Ms De Meyer told Belgium’s VRT News, adding: “The fact that this happens in two days is insane.”

Pia and her mother will fly to the US for treatment as soon as the money hits their account, which is 40 days.

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