rarerelated tag: Pediatrics/Child care

Dr. Richard S. Finkel is the Director of Center for Experimental Neurotherapeutics at St. Jude Children’s Research Hospital. Dr. Finkel has served as Chief of Neurology at Nemours Children’s Hospital in Orlando, and is the Professor of Neurology at the University of Central Florida College of Medicine. Dr. Finkel will also have privileges at Memphis‐based Le Bonheur Children’s Hospital and will have an adjunct faculty appointment in the Department of Pediatrics at the University of Tennessee Health Science Center. The three organizations will partner in the creation of a Neuroscience Research Consortium investigating treatments for children with devastating neuromuscular diseases.

 

Research Interests:

• Participation in clinical trials with investigational drugs for pediatric neurologic and metabolic diseases

• Development of outcome measures, biomarkers, and clinical trial design for pediatric neurologic diseases

• Understanding the trajectories of change in pediatric neuromuscular diseases: spinal muscular atrophy, Duchenne muscular dystrophy, and Charcot-Marie-Tooth inherited neuropathies

 

Representative Publications:

Treatment of Infantile-Onset Spinal Muscular Atrophy With Nusinersen: A Phase 2, Open-Label, Dose-Escalation Study

Nusinersen Versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Nusinersen Versus Sham Control in Later-Onset Spinal Muscular Atrophy

Onasemnogene Abeparvovec Gene Therapy for Symptomatic Infantile-Onset Spinal Muscular Atrophy in Patients With Two Copies of SMN2 (STR1VE): An Open-Label, Single-Arm, Multicentre, Phase 3 Trial