Betsy Malkus
Ultragenyx Pharmaceutical (United States)(US)
Publications by Year
Research Areas
Lysosomal Storage Disorders Research, Muscle Physiology and Disorders, Cerebral Palsy and Movement Disorders, Neonatal Respiratory Health Research, RNA Research and Splicing
Most-Cited Works
- → Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study(2015)213 cited
- → Tolerance of high-dose (3,000 mg/day) coenzyme Q10 in ALS(2005)152 cited
- → Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy(2016)72 cited
- → Pentoxifylline as a rescue treatment for DMD(2012)25 cited
- → Disease characteristics, early effectiveness, and safety of vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII) assessed in a novel, longitudinal, multicenter Disease Monitoring Program (DMP)(2021)1 cited
- → Performance measures and patient/caregiver-reported assessments collected in a longitudinal, multi-center disease monitoring program (DMP) of patients with mucopolysaccharidosis type VII (MPS VII)(2024)
- Clinical Diagnosis of Internal Diseases of Domestic Animals.(1937)
- → Disease characteristics, early effectiveness, and safety of vestronidase alfa for treatment of mucopolysaccharidosis VII assessed in novel disease monitoring program(2021)
- → Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in subjects with mucopolysaccharidosis type VII < 5 years old(2020)
- → Vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII): Updated results from a novel, longitudinal, multicenter disease monitoring program (DMP)(2022)