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Questions and Answers
What is the cardinal feature of Hereditary Angioedema (HAE)?
What is the cardinal feature of Hereditary Angioedema (HAE)?
- Recurrent attacks of subcutaneous swelling (correct)
- Recurrent attacks of muscle weakness
- Recurrent attacks of joint pain
- Recurrent attacks of visual disturbances
Which body sites have a predilection for swelling in Hereditary Angioedema?
Which body sites have a predilection for swelling in Hereditary Angioedema?
- Hands, feet, face, and genitals (correct)
- Abdomen and chest
- Head and neck
- Back and shoulders
What is the inheritance pattern of Hereditary Angioedema?
What is the inheritance pattern of Hereditary Angioedema?
- Autosomal recessive
- Autosomal dominant (correct)
- X-linked dominant
- Mitochondrial
Which type of HAE is characterized by low levels of C1 inhibitor protein?
Which type of HAE is characterized by low levels of C1 inhibitor protein?
What is the major mediator of swelling in C1 inhibitor deficiency?
What is the major mediator of swelling in C1 inhibitor deficiency?
What triggers an increased risk of attacks in patients with HAE?
What triggers an increased risk of attacks in patients with HAE?
What is the recommended strategy for treating all abdominal attacks and any airway attacks in HAE patients?
What is the recommended strategy for treating all abdominal attacks and any airway attacks in HAE patients?
Which on-demand treatment option for HAE involves a slow intravenous injection?
Which on-demand treatment option for HAE involves a slow intravenous injection?
'Kallikrein' is involved in which pathway that leads to swelling in C1 inhibitor deficiency?
'Kallikrein' is involved in which pathway that leads to swelling in C1 inhibitor deficiency?
'C1 inhibitor deficiency' results in increased activity of which mediator responsible for swelling?
'C1 inhibitor deficiency' results in increased activity of which mediator responsible for swelling?
Which treatment option in the text has a short half-life, high cost, and intravenous formulation, limiting its use?
Which treatment option in the text has a short half-life, high cost, and intravenous formulation, limiting its use?
What is the effect of Lanadelumab on attack frequency compared to placebo?
What is the effect of Lanadelumab on attack frequency compared to placebo?
Which drug is described as a small molecule inhibitor of pre-kallikrein and the only licensed oral prophylaxis option?
Which drug is described as a small molecule inhibitor of pre-kallikrein and the only licensed oral prophylaxis option?
What is the most common indication for the use of C1 inhibitor concentrate injections in the text?
What is the most common indication for the use of C1 inhibitor concentrate injections in the text?
Which treatment option is described as having gastro-intestinal side-effects in some patients and requires several weeks to reach full effect?
Which treatment option is described as having gastro-intestinal side-effects in some patients and requires several weeks to reach full effect?
What is the dosing frequency for Berotralstat, the small molecule inhibitor of pre-kallikrein?
What is the dosing frequency for Berotralstat, the small molecule inhibitor of pre-kallikrein?
Which drug mentioned in the text is an anti-sense inhibitor of pre-kallikrein and is in phase 3?
Which drug mentioned in the text is an anti-sense inhibitor of pre-kallikrein and is in phase 3?
What is the effect of Berotralstat on attack frequency compared to placebo?
What is the effect of Berotralstat on attack frequency compared to placebo?
What is the common side effect of Deucrictibant according to the text?
What is the common side effect of Deucrictibant according to the text?
Which drug treatment option mentioned is seeking approval according to the text?
Which drug treatment option mentioned is seeking approval according to the text?
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