Acute hepatic porphyria (AHP) is a very rare genetic disorder.
A drug has been developed to treat AHP.
This drug was tested in a clinical trial involving 94 patients from 18 countries.
The drug was given to 48 of the patients. The other 46 patients were a control group.
Comment on the design of this clinical trial.
(2)
(ii)
Each patient was given 2.5 mg of the drug per kg of body mass, once a month.
The drug is available as a solution with a concentration of 189 mg cm−3.
Calculate the volume of drug that was given each month to a patient with a body mass of 64 kg.
(2)
(iii)
Nausea was experienced by 27 % of the patients receiving this drug.
Calculate the number of patients who experienced nausea.
(2)
This drug is a double-stranded RNA molecule.
Complete the diagram to show the base sequence on the other RNA strand.
(3)
The synthesis of the haem component of haemoglobin involves several steps.
Each step in the synthesis of haem is catalysed by a different enzyme.
This drug works by interfering with the mRNA copies from the gene coding for one of these enzymes.
Explain how the action of this drug helps patients with AHP.
(3)
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