‘An answer to so many prayers’: Steinbach family receives medication approval from province to treat their dying son

A Steinbach family is feeling relief after a medication that can improve the quality of their son’s life was approved in Manitoba. Joseph Bernacki explains.

A family from Steinbach has some relief tonight after weeks of medical uncertainty. One of the medications they need to treat their five-year-old son was given Exceptional Drug Status by the province to improve his quality of life as he faces a terminal illness.

“This is an answer to so many prayers,” said Kayla Krentz Dupont, mother of her five-year-old son, Leo.

“There was the level of shock in it, right, where this has been something we’ve been working towards and we didn’t know if it was within reach.”

After weeks of Krentz Dupont and her network of supporters sending emails to the province to cover the expensive medication, Minister of Health, Seniors and Long-Term Care Uzoma Asagwara contacted the family.

The minister delivered word that the process for provincial approval for the drug Miglustat was approved and being expedited for her son. The drug is used to treat rare genetic disorders like Niemann-Pick disease type C, which five-year-old Leo was diagnosed with in June.

The disease is a rare terminal genetic disorder that leads to progressive neurological damage over time and affects his ability to walk, speak and use his arms and legs.

“Leo’s body does not metabolize the same way another person’s body does,” Krentz Dupont said. “It’s the storage of lipids in his liver and eventually brain that have these neurological effects.”

“The Dupont family is navigating what can only be described as a heartbreaking situation,” Asagwara said.

“I was able to call Kayla earlier today and share with her that I have approved the medication and that our department will work with her and her family every step of the way to Leo being able to start the medication as soon as possible.”

Ultimate goal to obtain second medication not approved by Health Canada

Krentz Dupont said it’s been an uphill battle to find care for her son based on his rare condition, saying this is one step in their medication journey.

“This has become my full-time job trying to secure care for my child, trying to secure medication for my child,” Krentz Dupont said. “Having a child that is ill is a nightmare for any parent. Having a child that is terminally ill is another level.”

It’s unclear when Leo Dupont will receive the medication, but it’s a step forward towards their second goal.

“Miglustat is required in order for us to swing for the fences, if you will,” Krentz Dupont said. “Our ultimate goal is to get Leo access to Miplyffa. Miplyffa is a drug that works synergistically with Miglustat.”

According to Krentz Dupont, the two drugs are used in combination to treat Niemann-Pick disease type C. Miplyffa, however, at this time is not approved for use in Canada.

“For a drug not to receive approval in Canada, it has to go through a number of approvals within Health Canada,” Krentz Dupont said. “The challenge here is that Leo doesn’t have time for the years that such approval takes.”

Mario Dupont said medication for his son is needed now more than ever.

“When your child has a progressive disease, you slowly see your child’s health deteriorate on a daily basis,” he said.

“We really want access to this medication so that Leo can live the best life that he can live while he’s here with us.”

Minister Asagwara said for other families to reach out to the province if they are having challenges navigating exceptional drug status for their medication here in Manitoba.

“I encourage all Manitobans that if they have concerns about a process to reach out directly to my office, make sure that I’m aware, and we’ll work with them to see how we can help,” Asagwara said.