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SMA1 Infant Life-Saving Zolgensma Therapy Unavailable in Canada

Author: John and Amanda Hanki(i) : Contact: Amanda Hanki, Mother of Harper: amandahanki@outlook.com - John Hanki, Father of Harper: jphanki@gmail.com

Published: 2020-09-15

Synopsis and Key Points:

Ground-breaking new drug Zolgensma that could stop progression of Spinal Muscular Atrophy Type 1 (SMA1) is the most expensive medication in the world.

Even if the Hankis were able to access Zolgensma for Harper, the drug costs $2.1 million USD ($2.8 million CAD) making it the most expensive medication in the world.

Harper needs the treatment now. Research has shown that the longer you wait the more irreversible damage this neuromuscular disease will have.

Main Digest

Imagine being told that your six-month-old daughter has a terminal disease with a life expectancy of less than two years. And then, learning that there is a ground-breaking new gene therapy that could save her, but it costs $2.8 million dollars Canadian, making it the world's most expensive drug ever produced.

This is the position that Edmonton parents Amanda and John Hanki find themselves in. Their daughter Harper was born on July 12, 2019 and at just six months old, was diagnosed with Spinal Muscular Atrophy Type 1 (SMA1).

SMA1 is a progressive, rare, genetic disease affecting 1 in 10,000 babies.

Babies like Harper, with SMA 1 (the most severe type) can't sit without support, and have trouble breathing, feeding and swallowing. Intellectual activity is normal and SMA1 patients are often observed to be unusually bright and sociable. But without treatment, SMA1, is likely to be fatal within the first two years of life.

Harper Hanki diagnosis.
Harper Hanki diagnosis.

Zolgensma

Amanda and John have just learned that Harper is a good candidate for a ground-breaking new gene therapy for SMA1 called Zolgensma. The single required dose of Zolgensma has been shown to alter the underlying genetic cause of SMA1 and may permanently stop the disease.

Zolgensma® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). It targets the genetic root cause of SMA with a one-time-only dose and replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene - (www.zolgensma.com).

Although the drug is not yet approved in Canada, Harper has received approval through the Special Access Program (SAP) to access this medication. Even if the Hankis were able to access Zolgensma for Harper, the drug costs $2.1 million USD ($2.8 million CAD) making it the most expensive medication in the world. This demonstrates the significant hurdles the family currently faces in dealing with their daughter's rare disease.

Harper needs the treatment now. Research has shown that the longer you wait the more irreversible damage this neuromuscular disease will have. Harper will no longer be eligible for this treatment once she is two years old - July 12, 2021.

Harper Hanki at her first birthday.
Harper Hanki at her first birthday.

GoFundMe Campaign

Parents, Amanda and John, together with their six-year-old son, Kohen and four-year-old daughter, Kinsley, and a broad community of supporters, have launched a GoFundMe campaign: My Hero Harper to raise the $2.8 million needed to pay for Harper's Zolgensma therapy. So far, the GoFundMe campaign has raised $192,851.

So far, through a number of fundraising efforts including the GoFundMe, thousands of people and small businesses have cumulatively contributed more than $250,000 toward Harper's treatment.

Previous initiatives have varied from silent online auctions on Instagram, province-wide bottle drives to $5 Fridays, video game tournaments, multi-summit hiking challenges, and even a personalized cocktail in a Banff National Park restaurant.

There are several upcoming fundraisers planned, such as a head-shaving event, additional online auctions, and a Sober October Challenge.

This is the most expensive medication in the world, but if 510,000 people donate only $5 - the cost of a cup of coffee - Harper's treatment would be attainable.

While fundraising goes on, the Hanki family fights for a healthy future for Harper by providing her with daily physiotherapy, a cough assist machine, and spinal injections to protect her muscles from further deterioration. Through determination and generosity, a growing network of supporters is doing everything in their power to save baby Harper's life.

Follow Harper Hanki for Information and Updates

(i)Source/Reference: John and Amanda Hanki. Disabled World makes no warranties or representations in connection therewith. Content may have been edited for style, clarity or length.

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