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Treating a patient with ADP porphyria through suppression of both hepatic and erythroid ALA production
Jul 13, 2026
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Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases
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Likelihood-based calibration improves the clinical utility of JAG1 functional data for variant classification
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Clinical, pathological, and genetic characteristics of 23 DMD patients in northern China
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