Publications by authors named "W C Wilcox"
Background: Vosoritide is a C-type natriuretic peptide analog that addresses an underlying pathway causing reduced bone growth in achondroplasia. Understanding the vosoritide treatment effect requires evaluation over an extended duration and comparison with outcomes in untreated children.
Methods: After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.
Measurement of lysosomal disease (LD) biomarkers can reveal valuable information about disease status. Lyso-globotriaosylceramide (lyso-Gb), glucosylsphingosine (lyso-Gb), galactosylsphingosine (psychosine), and glucose tetrasaccharide (Glca1-6Glca1-4Glca1-4Glc, Glc) are biomarkers associated with Fabry, Gaucher, Krabbe, and Pompe disease, respectively. Clinical biomarker testing is performed to guide patient management, including monitoring disease progression and initiating treatment, and in diagnostic evaluations of either symptomatic patients or asymptomatic individuals with a positive family history or abnormal newborn screen.
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- Pegunigalsidase alfa is a new enzyme replacement therapy for Fabry disease, showing a longer half-life and administered every 4 weeks instead of the typical 2-week schedule.
- The BRIGHT study involved 30 adult patients who switched from another ERT to pegunigalsidase alfa, revealing good safety results with mostly mild side effects and no new anti-drug antibodies.
- Although the treatment showed acceptable tolerance, more research is needed due to the small sample size, but it suggests that this 4-week regimen could be a viable new option for managing Fabry disease.
A new bidentate bridging ligand, bis(2-pyridyl)thieno[3,4-]pyrazine is reported, along with its mono- and bi-metallic Ru(II) complexes as representative examples. Spectroscopic, electrochemical and X-ray crystallographic characterization of these species is reported, with the separation of the two Ru(III)/Ru(II) couples of the bimetallic complex suggesting better metal-metal communication than classical polypyridyl analogues.
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- The study aimed to assess how vosoritide affects the quality of life in children with achondroplasia over a 3-year period.
- Participants, aged around 9.7 years, received the treatment for an average of 4 years, following a prior placebo-controlled trial.
- Results showed significant improvements in physical and social health scores, especially in children who experienced notable height increases, indicating that vosoritide positively impacts their overall functioning.