A type of treatment developed for people with inhibitors. These treatments go around (or bypass) the clotting factors that are blocked by the inhibitor to help the body form a clot.
A research study involving human volunteers (also called participants) intended to add to the drug discovery and development process. They play a key role in evaluating potential new medicines, including their safety and how well they work.
Within this website, it is defined as making emicizumab-kxwh available on a case-by-case basis to an eligible person following a request from their treating physician. These requests are for people who have a serious or life-threatening condition, and have exhausted all other treatment options and are unable to participate in a clinical trial.
Within this website, it is defined as making emicizumab-kxwh available to intermediate to large populations who meet specific criteria in accordance with local health authority requirements (eg, the FDA). The medication is made available 1) outside of a clinical trial and 2) before its been approved.
Provides information about a clinical trial, its purpose, potential risks and benefits and the potential participant’s rights to allow for an informed decision about participation in the clinical investigation.
Medication guides are required by the FDA for certain prescribed medicines when the FDA determines that certain information is necessary to prevent serious side effects, patient decision-making should be informed by information about a known serious side effect with a product, or patient adherence to directions for the use of a product are essential to its effectiveness.
Generally refers to the time after a medication has been approved by the FDA. See FAQ for additional information.
Include studies and clinical trials that sponsors are required by the FDA to conduct under one or more statutes or regulations.
Risk-mitigation – Taking steps to minimize or eliminate harm or loss.
Thrombotic microangiopathy (TMA)
This is a condition involving blood clots and injury to small blood vessels that may cause harm to your kidney, brain, and other organs.
A blood clot that may form in blood vessels in an arm, leg, lung, or head.
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