Edward M. Kaye
Spero Therapeutics (United States)(US)
Publications by Year
Research Areas
Lysosomal Storage Disorders Research, Muscle Physiology and Disorders, Metabolism and Genetic Disorders, Mitochondrial Function and Pathology, Cellular transport and secretion
Most-Cited Works
- → Eteplirsen for the treatment of Duchenne muscular dystrophy(2013)778 cited
- → Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy(2011)548 cited
- → Longitudinal effect of eteplirsen versus historical control on ambulation in D uchenne muscular dystrophy(2015)497 cited
- → Natural history of infantile‐onset spinal muscular atrophy(2017)401 cited
- → Clinical research for rare disease: Opportunities, challenges, and solutions(2008)398 cited
- → Childhood ataxia with diffuse central nervous system hypomyelination(1994)263 cited
- → A Controlled Trial of Diazepam Administered during Febrile Illnesses to Prevent Recurrence of Febrile Seizures