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Keyphrases
Phase 1 Trial
100%
Acute Intermittent Porphyria
100%
RNA Interference Therapy
100%
Givosiran
100%
Placebo
62%
5-aminolevulinate Synthase
50%
Adverse Events
25%
Messenger RNA (mRNA)
25%
Two-dose
25%
Porphobilinogen
25%
Attack Rate
25%
Porphyria
25%
5-aminolevulinic Acid (5-ALA)
25%
Abdominal Pain
12%
Serious Adverse Events
12%
Therapeutic Agents
12%
Gene Expression
12%
MRNA Level
12%
Diarrhea
12%
Subcutaneous Injection
12%
Disease Manifestations
12%
Heme Biosynthesis
12%
Event Reduction
12%
Recurrent Attacks
12%
RNA Therapeutics
12%
Nasopharyngitis
12%
Gene Accumulation
12%
Rare Inherited Disease
12%
Efficacy Outcomes
12%
Pharmacokinetic/pharmacodynamic (PK/PD)
12%
Safety Pharmacokinetics
12%
Ascending Dose
12%
Nursing and Health Professions
Acute Intermittent Porphyria
100%
Givosiran
100%
RNAi Therapeutics
100%
Injection
87%
Placebo
62%
5 Aminolevulinate Synthase
50%
Adverse Event
37%
Porphyria
25%
Aminolevulinic Acid
25%
Messenger RNA
25%
Efficacy
25%
Porphobilinogen
25%
Body Weight
12%
Randomization
12%
Heme
12%
Diarrhea
12%
Abdominal Pain
12%
Genetic Disorder
12%
Subcutaneous Injection
12%
Pharmacodynamics
12%
Rhinopharyngitis
12%
Diseases
12%
Accident Prevention
12%
Pharmacology, Toxicology and Pharmaceutical Science
Acute Intermittent Porphyria
100%
Givosiran
100%
Placebo
62%
5 Aminolevulinate Synthase
50%
Adverse Event
37%
Aminolevulinic Acid
25%
Porphyria
25%
Porphobilinogen
25%
Messenger RNA
25%
Pharmacodynamics
12%
Subcutaneous Injection
12%
Abdominal Pain
12%
Genetic Disorder
12%
Diarrhea
12%
Heme
12%
Rhinopharyngitis
12%
Pharmacokinetics
12%
Diseases
12%
Neuroscience
Porphyria
100%
RNAI
100%
Givosiran
100%
Placebo
62%
5 Aminolevulinate Synthase
50%
Aminolevulinic Acid
25%
Porphobilinogen
25%
Messenger RNA
25%
Gene Expression
12%
Anabolism
12%
Heme
12%
Pharmacokinetics
12%
Biosynthesis
12%
Biochemistry, Genetics and Molecular Biology
Porphyria
100%
RNAI
100%
Aminolevulinic Acid Synthase
80%
Messenger RNA
40%
Porphobilinogen
40%
Body Weight
20%
Anabolism
20%
Gene Expression
20%
Genetic Disorder
20%
Pharmacokinetics
20%
Pharmacodynamics
20%
Mathematics
Exploratory
100%
End Point
50%
Randomization
50%