Not only can access to medicine mean life-changing results for those living with a rare disease, it can also make a world of difference for loved ones such as parents, siblings and friends. In our next installment of Advocacy in Action, parents from B.C. explain why it has been so important to them that their daughter has gained access to OrkambiTM.
“We were recently approved for access to OrkambiTM on compassionate basis for our 14-year-old daughter. Our daughter has been ill for some time with a highly resistant form of Mycobacterium Abscessus infection with associated weight loss, periodic hemoptysis, consistently positive cultures, sustained drop in lung function, cough, and telltale damage visible on her chest CT scan. The prognosis for CF patients with this infection is poor, even with aggressive treatment. She is about to start an intensive 18 month treatment course (the first treatment course failed), which is why we wanted to give her the best chance possible with the addition of OrkambiTM.
“Remarkably, six weeks after starting OrkambiTM, our daughter had gained a much-needed 10lbs – a feat she could only otherwise accomplish with 24/7 tube feeds, which is very stressful for her. Her cough has decreased, and so far she has not had any further episodes of hemoptysis. The weight gain in itself is extremely important as it’s directly correlated with increased life expectancy and will surely help her to have the reserves needed to fight off this infection.”
OrkambiTM: Offering Hope
“It means so much to us as parents to know that with the seriousness of our daughter’s condition, we are doing everything in our power to give her the best chance of surviving this ordeal and having a happy, healthy life. OrkambiTM is state-of-the-art precision medicine for CF patients with our daughter’s specific genetic mutation and we are so grateful that she has the opportunity to benefit from it. We are very hopeful that by treating the CF defect at its most basic level, OrkambiTM will significantly enhance the effectiveness of the grueling treatment that she is about to undertake and give her a better chance for a bright future.”
For more information on our advocacy efforts, visit cysticfibrosis.ca/advocacy/raising-our-voice.