William Ross Wilcox

机构：Emory University · ORCID：0000-0002-0325-0377

发表论文 561 篇 · 总被引 23503 次 · h-index 72

代表论文

• Vosoritide therapy in children with achondroplasia aged 3−59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial (2023 · The Lancet Child & Adolescent Health · 被引 68)
• Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study (2022 · Molecular Genetics and Metabolism · 被引 55)
• Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study (2023 · Journal of Medical Genetics · 被引 49)
• A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA-α5 NAM (basmisanil) on intellectual disability associated with Down syndrome (2022 · Journal of Neurodevelopmental Disorders · 被引 48)
• Global reach of over 20 years of experience in the patient-centered Fabry Registry: Advancement of Fabry disease expertise and dissemination of real-world evidence to the Fabry community (2023 · Molecular Genetics and Metabolism · 被引 37)
• Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes (2022 · Journal of Medical Genetics · 被引 37)