Publications by authors named "Julia M Migliorati"
Article Synopsis
- There is a critical need for effective drug therapies for the genetic disorder primary hyperoxaluria (PH), which includes three subtypes: PH1, PH2, and PH3, characterized by oxalate accumulation leading to kidney stones.
- Currently, nedosiran, an siRNA-based drug, is being developed to treat all three PH types, and it has shown promise in clinical trials by significantly reducing urinary oxalate levels in patients with PH1.
- Clinical trials have also indicated that nedosiran has a favorable safety profile, with mostly mild injection site reactions and no severe adverse events reported, making it a strong candidate for treating PH.
Absorption, distribution, metabolism, and excretion (ADME) are the key biologic processes for determination of a drug's pharmacokinetic parameters, which have direct impacts on efficacy and adverse drug reactions (ADRs). The chemical structures, dosage forms, and sites and routes of administration are the principal determinants of ADME profiles and consequent impacts on their efficacy and ADRs. Newly developed large molecule biologic antisense oligonucleotide (ASO) drugs have completely unique ADME that is not fully defined.
View Article and Find Full Text PDFThere is large interindividual variability in the efficacy of pre-exposure prophylaxis (PrEP) with tenofovir (TFV) in preventing HIV infection. Naturally occurring mutations in the creatine kinase M-type (CKM) gene examined by Mosher et al. could provide answers to why some individuals who have active serum creatine kinase in clinical assessments may not respond to TFV PrEP.
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