How does hemophilia affect hemostasis?
Hemophilia is caused by a mutation or change, in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot. This change or mutation can prevent the clotting protein from working properly or to be missing altogether.
How does hemophilia affect body systems?
Hemophilia can result in: Bleeding within joints that can lead to chronic joint disease and pain. Bleeding in the head and sometimes in the brain which can cause long term problems, such as seizures and paralysis. Death can occur if the bleeding cannot be stopped or if it occurs in a vital organ such as the brain.
How does hemophilia B affect the body?
In severe cases of hemophilia B, frequent, spontaneous bleeding episodes are the most common symptom. Spontaneous bleeding episodes may include bleeding into the muscles and joints. This often causes pain and swelling and restricts movement of the joint. Bleeding into a joint is called a hemarthrosis.
What part of the cell does hemophilia affect?
Platelets are small blood cells that form in your bone marrow. According to the World Federation of Hemophilia (WFH), about one in 10,000 people are born with this disease. People with hemophilia bleed easily, and the blood takes a longer time to clot.
Does Trikafta increase life expectancy?
Life expectancy is on the rise According to the 2018 Cystic Fibrosis Foundation (CFF) Patient Registry, people with CF born between 2014 and 2018 have a predicted life expectancy of 44 years. However, while available data is currently limited, Trikafta is expected to increase life expectancy even more.
Is Trikafta a cure for CF?
The F508del mutation is the most common CF-causing genetic defect, seen in about 90% of all people with the disease. In December 2020, the U.S. regulatory agency expanded the number of approved mutations for Trikafta, meaning the therapy can now be used to treat a broader range of CF patients.
Does Trikafta make you gain weight?
This drug was everything I had hoped for. Along with all the positive lung-related improvements, I also began to gain weight. My CF care team had said from the beginning that weight gain was experienced by people on Trikafta. I have been about the same weight within a few pounds for the past seven-to-eight years.
What age is Trikafta for?
The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children ages 6 through 11 who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.
What are the side effects of Trikafta?
TRIKAFTA may cause serious side effects including increased liver enzymes and clouding of the lens in the eye (cataracts). The most commonly reported side effects associated with TRIKAFTA are headache, upper respiratory infections, abdominal pain, diarrhea, rash and elevated liver enzymes.
Who created Trikafta?
Developed by Vertex Pharmaceuticals, the new drug application (NDA) for TRIKAFTA was accepted for review and granted priority review status by the US Food and Drug Administration (FDA) in August 2019. The FDA approved the drug in October 2019.
When was Trikafta FDA approved?
FDA Approves Trikafta to Treat Children Starting at Age 6 Use of the triple combination therapy, first approved in the U.S. in 2019, was previously limited to CF patients ages 12 and older with these mutations.
Can you drink alcohol on Trikafta?
Do not drive, use machinery, or do anything that needs alertness until you can do it safely. Limit alcoholic beverages.
What class of drug is Trikafta?
Trikafta belongs to a class of drugs called CFTR modulators. It is designed for people with cystic fibrosis (CF) who have at least one of 178 different mutations in their CFTR (cystic fibrosis transmembrane conductance regulator) gene. This includes the most common mutation, F508del.