StemCell Therapy

An Edmonton babys parents are trying to raise $2.8 million to pay for a gene therapy treatment to stop her rare neurodegenerative disorder before her second birthday.

Harper Hanki was diagnosed Spinal Muscular Atrophy Type 1 in January 2020. Its a terminal genetic disorder that will see Harper gradually lose control of her muscular movements.

Shes very vocal. Happy all the time. Determined,her father John Hanki told Daily Hive.

Her disorder is the same one that two Metro Vancouver babies also have. Baby Lucy and baby Aryan made headlines earlier this summer as their families both successfully raised the nearly $3 million needed to import the lifesaving drug from the US.

The Hanki family is incredibly happy for those families, and hopes their own fundraising journey isnt old news.

The drug that will help Harper is called Zolgensma. Its one-time injection that will insert a working copy of Harpers defective gene, effectively stopping the degenerative disorder in its tracks.

But theres a catch: Zolgensma the most expensive drug in the world. The treatment is sold by Swiss drug-maker Novartis, and it isnt yet approved in Canada. That means its not covered by any provincial health plan, and families like the Hankis have to import the drug from the US through their local hospital.

Time is of the essence, because Zolgensma is only effective in children under two years old. Harper just turned one, and her parents hope to get the treatment as soon as possible.

So far, theyve raised $152,000 through their GoFundMe page.

Even though thats an absurd amount of money, its just hard to know that its not even close, Amanda Hanki, Harpers mother, told Daily Hive. For the circumstances, its unfortunately just a drop in the bucket.

So the family has produced a video telling Harpers story and asking for donations from friends, family, and complete strangers.

We know its possible You see the Oilers 50/50 get to $15 million in seven hours, were just thinking how do we get our hands on that, Amanda said.

For now, Harper is getting injections of Spinraza. Its a temporary solution that slows SMAs progress. As each injection wears off, John says its clear Harper is gradually losing motor function.

She cant really move like a one-year-old should, he said.

The Hankis are in talks with their local MLA to try and lobby Alberta Health Minister Tyler Shandro to include Zolgensma under the provinces public health coverage.

Although SMA is a rare disease, the Hankis know Harper wont be the last baby born with it.

Link:
Baby Harper races to raise $2.8 million for gene therapy treatment | News - Daily Hive

This entry was posted on August 24, 2020 at 9:56 pm and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |