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ADVERSE REACTIONS In adult patients, the most frequently reported adverse reactions among CRESEMBA-treated patients were nausea (26%), vomiting (25%), diarrhea (22%), headache (17%), elevated liver chemistry tests (16%), hypokalemia (14%), constipation (13%), dyspnea (12%), cough (12%), peripheral edema (11%), and back pain (10%)

During the trial design phase, digital approaches such as machine learning algorithms and digital twin models enable precise cohort selection and the generation of predictive models to optimize sample sizesan essential feature for rare diseases with limited patient pools

Ipamorelin: A Selective GHRP Peptide Ipamorelin is a growth hormone-releasing peptide (GHRP) that stimulates GH secretion without significantly increasing cortisol or prolactin levels

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