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Rare Diseases (RD)
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Rare Diseases (RD)
Rare Diseases (RD)
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PT-024 - EVIDENCE SUPPORTING EFFICACY EXTRAPOLATIONS FOR TARGETED THERAPIES ACROSS MOLECULARLY DEFINED SUBSETS OF DISEASES IN THE ABSENCE OF SUBSET-SPECIFIC CLINICAL DATA.
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PII-179 - A COMPUTATIONAL TOOL TO OPTIMIZE CLINICAL TRIAL DESIGN FOR DUCHENNE MUSCULAR DYSTROPHY: A PRACTICAL GUIDE AND CASE STUDIES.
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PII-180 - CLINICAL PHARMACOLOGY CONSIDERATIONS FOR FIRST-IN-HUMAN CLINICAL TRIALS FOR ENZYME REPLACEMENT THERAPY
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PII-181 - FLUOROQUINOLONES ATTRIBUTE AORTIC DISEASES THROUGH ENDOTHELIAL DYSFUNCTION.
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PII-182 - POPULATION PHARMACOKINETICS (PK) AND PK/PHARMACODYNAMICS ANALYSES TO REFINE PHASE 3 DOSE OF SETRUSUMAB IN PEDIATRIC PATIENTS WITH OSTEOGENESIS IMPERFECTA: RESULTS FROM PHASE 2 OF THE ORBIT STUDY
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PII-183 - POPULATION PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-07265803 AND ITS METABOLITE IN PATIENTS WITH LAMIN A/C RELATED DILATED CARDIOMYOPATHY.
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PII-184 - TOWARDS ASSISTING POTENTIAL PREVENTION CLINICAL TRIALS OF BONE-PROTECTIVE THERAPIES FOR DUCHENNE MUSCULAR DYSTROPHY USING DEEP SEGMENTED IMAGING BIOMARKER
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PWI-004 - EVIDENCE SUPPORTING EFFICACY EXTRAPOLATIONS FOR TARGETED THERAPIES ACROSS MOLECULARLY DEFINED SUBSETS OF DISEASES IN THE ABSENCE OF SUBSET-SPECIFIC CLINICAL DATA.
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