Betsy C. Malkus

机构：Ultragenyx Pharmaceutical (United States)

发表论文 18 篇 · 总被引 499 次 · h-index 5

代表论文

• Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study (2015 · Neurology · 被引 231)
• Tolerance of high-dose (3,000 mg/day) coenzyme Q10 in ALS (2005 · Neurology · 被引 154)
• Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy (2016 · The American Journal of Human Genetics · 被引 75)
• Pentoxifylline as a rescue treatment for DMD (2012 · Neurology · 被引 25)
• Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII (2022 · Molecular Genetics and Metabolism · 被引 7)
• Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program (2024 · Orphanet Journal of Rare Diseases · 被引 4)