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Welcome to BBT, a trust founded in 2018 by a local South African family who has been affected by cystic fibrosis (CF) in South Africa. Their mission is to ensure that Lize, a patient affected by CF, has enough funds to live a healthy and normal life. The dream is to provide access to expensive yet life-saving medications and treatments for not only Lize, but to all patients who need it, and who needs it now.
Cystic fibrosis (CF) is a genetic disorder that primarily affects the respiratory and digestive systems. There is currently no cure for cystic fibrosis, and treatment mainly focuses on managing symptoms, preventing complications, and improving quality of life. This can include medications to help clear the airways, prevent infections, and improve digestion, as well as physical therapy and other supportive measures.
In South Africa, access to these treatments is limited, and many families struggle to afford the high cost of care. This is where BBT comes in. BBT was founded by a local family who knows firsthand the impact that CF can have on a person's life and the lives of those around them. Our main beneficiary, Lize de Villiers, was born on 3 November 2000, and was diagnosed at the age of 3 months. Yet, her parents were told that she would not live to see the day she turned 10. BBT is initiated to help support Lize and her family, but also with the ultimate goal to extend this support to others who are financially needy. As a parent, your only wish is to see your children thrive and live the life they've always dreamed of. BBT was the dream of one such parent to ensure her daughter has enough funds to take care of herself and to live without boundaries.
One such medication, the newest so-called 'miracle-drug', is called Trikafta. Trikafta is considered an important drug for people with cystic fibrosis as it is the first treatment that addresses the underlying cause of the disease in most people with the condition. It is the first medication that targets the F508del mutation, the most common genetic mutation of cystic fibrosis, which accounts for about 90% of the cases worldwide. Trikafta works by correcting the CFTR protein function and helps to clear the mucus from the lungs, reducing inflammation and preventing infections. This improves lung function, reducing the need for hospitalizations, and improving the quality of life for people with cystic fibrosis. Overall, Trikafta is a significant step forward in the treatment of cystic fibrosis and is considered a game-changer for people living with this condition. It is a hope for a better life for patients and a way to reduce the burden on the healthcare system.
However, Trikafta is not yet available in South Africa because it has not yet been approved by the regulatory authorities in the country due to the expensive nature of the drug. The process of approving new medications in South Africa can be lengthy and complex, involving rigorous safety and efficacy assessments, and can take several years to complete. Besides this, the medication was priced in 2021 at approximately $311,000 USD per year. Only a handful of patients in 1st world countries have access, and the manufacturers are not helping the community. See https://www.vertexsaveus.org/ for more information and to join the fight against this injustice.
BUT THERE IS HOPE!
Trixacar, a generic version of Trikafta was developed by Gador in Argentinia. Although South Africans are not legally allowed to import this generic medication due to patent laws, they are allowed to fly to Argentinia and collect a few months' supply. As one could imagine, although this is a feasible option, it is only feasible for those who can afford doing so.
South Africans cannot legally import this medication due to patent laws, but they can fly to Argentina to collect a few months' supply. Lize started taking Trixacar in March 2022 and experienced significant improvements in her health and quality of life. Within a few days or weeks of starting Trikafta, CF patients have noticed improved lung function and easier breathing. This can lead to increased energy levels, less fatigue, and better overall health. Trikafta has been shown to improve lung function, reduce exacerbations and hospitalizations, and improve digestion and weight gain. Lize's doctor was amazed at her improvements and both were close to tears realizing the impact the medicine has on her life. The price of Trixacar is still high and unstable, but stopping it is not an option for those who have experienced its benefits.
We therefore aim to help fund the cost of expensive medications, treatments and equipment needed to manage the symptoms of CF. The money currently goes directly into treatments and medication for Lize, so that she has a reliable and sustainable, normal, future ahead of her.
Help Lize to continue using this medication and ensure a quality of life that was never before promised. We believe in transparency and will deliver regular updates in the Blog section.