Neurology Research

The Division of Child Neurology at UPMC Children’s Hospital of Pittsburgh is at the frontier of medical research. Over the past decade, neurological science has made major discoveries in understanding the genetic, molecular, cellular and organizational basis for diseases of the brain, spinal cord, nerves and muscles. These advances have improved our ability to diagnose and treat many previously incurable disorders of the nervous system.

Children’s Hospital’s neurologists are leaders in research. Faculty members of the Division of Child Neurology bring into the Pittsburgh biomedical community in excess of $1 million in federal corporate and foundation research funding each year. Their research studies range from determining the best seizure medications to use for particular epilepsies to creating targeted viruses to treat cancer.

Basic science and clinical investigations directly related to our mission are an integrated component of the division.

Children’s neurologists are equally at home and accomplished in the classroom. All teach at the University of Pittsburgh School of Medicine. Beyond the lecture hall and conference room, they also are career mentors for high school, undergraduate and medical students, residents and fellows from all over the country. They have also hosted international colleagues in observerships in child neurology.

Researchers

Clinical Studies

Childhood Absence Epilepsy Study

Childhood Absence Epilepsy: Rx PK-PD-Pharmacogenetics Study

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Cough Study for Males with Neuromuscular Disease – Pilot

Cough Peak Flow And Airway Clearance in Pediatric Patients with Neuromuscular Disease

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Dravet Syndrome Treatment with Lorcaserin – Phase III

Study with Open-Label Extension Phase of Lorcaserin as Adjunctive Treatment in Subjects with Dravet Syndrome

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Duchenne Muscular Dystrophy and MP-104

Expanded Access Protocol Intended to Provide Treatment With MP-104 (deflazacort) to U.S. Children, Adolescents, and/or Adults with Duchenne Muscular Dystrophy

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Duchenne Muscular Dystrophy and SRP-4045 and SRP-4053 – Phase III

Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with Duchenne Muscular Dystrophy

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Duchenne Muscular Dystrophy and Treatment With PF-06252616 – Phase II

Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of PF-06252616 in Ambulatory Boys With Duchenne Muscular Dystrophy

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Duchenne Muscular Dystrophy Treatment With Ataluren – Phase III

Study Of Ataluren (PTC124) In Patients With Nonsense Mutation Dystrophinopathy

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Duchenne Muscular Dystrophy Treatment With Eteplirsen – Phase III

Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy

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Epilepsy Rescue Therapy with USL261 Spray: The ARTEMIS-1 Study – Phase III

Study of the Safety and Efficacy of Intranasal Midazolam (USL261) in the Outpatient Treatment of Subjects with Seizure Clusters

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Epilepsy Rescue Therapy with USL261 Over a Long-Term – Phase III

Study of USL261 in the Outpatient Treatment of Subjects with Seizure Clusters

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Epilepsy Surgery Study

Retrospective Review of Pediatric Epilepsy Surgery Program

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Epilepsy Therapy Using Brivaracetam with Other Medications – Phase III

Study to Evaluate the Safety and Efficacy of Brivaracetam Used as Adjunctive Treatment in Pediatric Subjects with Epilepsy

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Establishing Cytokine Profiles in Patients with MS

Cytokine Profiles in Pediatric Multiple Sclerosis and Other Acute Demyelinating Disorders in Children

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Migraine Treatment with Galcanezumab: The REBUILD-1 Study – Phase III

Study of Galcanezumab in Adolescent Patients 6 to 17 Years of Age with Episodic Migraine – the REBUILD-1 Study

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Migraine Treatment with Galcanezumab: The REBUILD-2 Study – Phase III

Study of Galcanezumab in Adolescent Patients 12 to 17 Years of Age with Chronic Migraine – the REBUILD-2 Study

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Saliva Test for Anti-Seizure Drug Levels in Infants

Phenobarbital Levels in Neonates: A Comparison Between Blood and Saliva Levels

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Stroke Study and Database

Institutional and International Pediatric Stroke Study and Database

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