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Researchers

Melissa Spencer, PhD

Researcher
Co-Director
Center for Duchenne Muscular Dystrophy, UCLA
635 Charles Young Dr. So
Los Angeles, California, United States

Dr. Melissa Spencer is a Professor of Neurology at UCLA. She did her PhD and post-doctoral studies at UCLA, elucidating pathogenic mechanisms of Duchenne Muscular Dystrophy. As faculty member in the Department of Neurology, Dr. Spencer’s research program continues to focus on pathogenesis and therapies for Duchenne and Limb Girdle Muscular Dystrophies, using a longitudinal and multidisciplinary approach that ranges from the generation of genetically modified mice to use of biochemical, cell biological, immunological and functional approaches for analyzing phenotypic outcomes.

She has led two clinical trials at UCLA, with Dr. Fowler as co-investigator. The trials assessed the efficacy of Albuterol in Duchenne and provided much of the framework and clinical trial structure that currently exists at UCLA. This foundation has enabled UCLA’s participation in industry and investigator initiated trials for Duchenne. Dr. Spencer was the recipient of a Presidential Early Career Award for Scientists and Engineers (PECASE).

 

Representative Publications:

Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy

Emerging Genetic Therapies to Treat Duchenne Muscular Dystrophy

Albuterol Increases Lean Body Mass in Ambulatory Boys With Duchenne or Becker Muscular Dystrophy

Pilot Trial of Albuterol in Duchenne and Becker Muscular Dystrophy

 

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