2023-09-09 18:30:17
A baby, just 9 days old, who had spinal muscular atrophy disease is the youngest patient in North America to receive $2.4 million worth of medication to prevent further harm. develop symptoms.
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Little Samuel’s sister, 9 years old, suffers from this progressive degenerative disease with devastating consequences on the ability to breathe, eat or even walk.
She still has the after-effects, despite the clinical trial received in the United States at the age of six months.
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Samuel had a one in four chance of developing the same disease which, a few years ago, could be fatal after a year.
The medical team at the Montreal Children’s Hospital did not want to take any risks and approved a very expensive gene therapy for Samuel, just nine days after his birth.
Tests were carried out when Samuel’s mother was pregnant and these suggested that he could have the rare disease.
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Four months later, the toddler has no symptoms associated with spinal muscular atrophy, rejoices his doctor.
The costs associated with the medication have been fully reimbursed by RAMQ since 2021.
“He would not be seated, he would not have control of his trunk, he would have lost his reflexes, he would have more difficulty breathing, he would no longer be able to eat at that age. We know that her sister had difficulty swallowing, she choked at that age,” explains Dr. Myriam Oskoui, neurologist at the hospital center associated with McGill University.
Although she would have liked her daughter to benefit from the same medication her son received, Samuel’s mother is delighted to see medical progress in the fight against spinal muscular atrophy.
She admits that her daughter’s medical condition literally saved her son’s life.
“It’s like a bittersweet feeling, let’s put it that way. For Arianne, we were so ready, but she is still in the generation where there wasn’t much. But now, we know that thanks to it, children today will not experience the negative effects of this disease by being treated and detected earlier. He will not experience that of having his muscles that do not work, of not being able to walk, of not being able to sit,” explains Audrey Bouchard in an interview with TVA Nouvelles.
Since 2021, at the Montreal Children’s Hospital, 21 patients have received Zolgensma, considered one of the most expensive drugs in the world.
*See Harold Gagné’s explanations in the video above*
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