Pediatrics, Neurology

• 

  People

  • Administrative Positions

    • Mikati, Mohamad Abdul, Chief, Division of Pediatric Neurology

  • Professorships

    • Mikati, Mohamad Abdul, Wilburt C. Davison Distinguished Professor

  • Primary Appointments

    • Gospe Jr., Sidney M., Adjunct Professor in the Department of Pediatrics
    • Jasien, Joan Mary, Associate Professor of Pediatrics
    • Kansagra, Sujay Mansukhlal, Associate Professor of Pediatrics
    • Kenney-Jung, Daniel Lewis, Assistant Professor of Pediatrics
    • Lachiewicz, Ave Maria, Associate Professor Emeritus of Pediatrics
    • Lemmon, Monica Elizabeth, Associate Professor of Pediatrics
    • Lewis Jr., Darrell Vincent, Professor Emeritus of Pediatrics
    • Mikati, Mohamad Abdul, Professor of Pediatrics
    • Patel, Shital, Assistant Professor of Pediatrics
    • Pizoli, Carolyn Elizabeth, Assistant Professor of Pediatrics
    • Rathke, Kevin Marc, Clinical Associate in the Department of Pediatrics
    • Russ, Jeffrey Brian, Medical Instructor in the Department of Pediatrics
    • Salter, Alice Bryn, Assistant Professor of Pediatrics
    • Smith, Edward Clinton, Professor of Pediatrics
    • Tchapyjnikov, Dmitry, Adjunct Assistant Professor in the Department of Pediatrics
    • Valdez, Purnima Thyagarajan, Associate Professor of Pediatrics
    • Werner, Klaus-Georg Erich, Assistant Professor of Pediatrics
    • Worley, Gordon, Professor Emeritus of Pediatrics
    • Zafar, Muhammad Shahzad, Assistant Professor of Pediatrics
• 

  Administers Grant

  • 1042-TSC-2001 - Marinus TSC
  • 2019 PPMD CDCC Grant
  • 2020-2025 PPMD CDCC Grant
  • 2021 PPMD CDCC Grant
  • 2022 PPMD CDCC Grant
  • A Comprehensive Newborn Screening Solution for Duchenne and Congenital Muscular Dystrophies
  • A Double-Blind, Placebo-Controlled, Randomized-Withdrawal, Multicenter Study of the Efficacy and Safety of Xyrem with an Open-Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects with Narcolepsy with Cataplexy
  • A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Children and Young Adults with Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Followed by Long-term Open-label Treatment
  • A Learning Healthcare System for Pediatric Epilepsy
  • A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
  • A Long-term Observational Study Evaluating Eteplirsen in Patients with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice
  • A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated with Epidiolex (Cannabidiol) Oral Solution
  • A MULTICENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF LACOSAMIDE AS ADJUNCTIVE THERAPY IN SUBJECTS WITH EPILEPSY >1 MONTH TO <4 YEARS OF AGE WITH PARTIAL-ONSET SEIZURES
  • A MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY, AND TOLERABILITY OF INTRAVENOUS BRIVARACETAM IN SUBJECTS ¿1 MONTH TO <16 YEARS OF AGE WITH EPILEPSY PHASE 2
  • A MULTICENTER, OPEN-LABEL, LONG-TERM EXTENSION STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF LACOSAMIDE AS ADJUNCTIVE THERAPY IN PEDIATRIC SUBJECTS WITH EPILEPSY WITH PARTIAL-ONSET SEIZURES PHASE 3
  • A Phase 2 Study to Assess the Safety, Tolerability, Exploratory Efficacy, and Pharmacokinetics of Orally Administered JBPOS0101 for Refractory Infantile Spasms Patients
  • A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive
  • A Phase 3 Multinational, Randomized, Double-Blind, Placebo Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP 9001 in Subjects With Duchenne Muscular Dystrophy" ("Study"); Sarepta Protocol Number SRP-9001-301
  • A Phase I, Open-Label, Pharmacokinetic, Dose-Escalation Study Of Cenobamate (YKP3089) In Pediatric Subjects With Partial Onset Seizures
  • A Phase II , Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)
  • A Randomized, Double-Blind, Placebo- Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Lab
  • A Retrospective Multicenter Study to Investigate Dosage, Efficacy,and Safety of Fycompa® in Routine Clinical Care of Patients With Epilepsy
  • A Two-Part Study of ZX008 in Children and Adults with Lennox-Gastaut Syndrome (LGS)
  • A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment
  • AN OPEN-LABEL, SINGLE-DOSE, PHARMACOKINETICS STUDY OF VALTOCO® WITH OPEN-LABEL SAFETY PERIOD IN PEDIATRIC SUBJECTS WITH EPILEPSY
  • ATPA2 Research Project
  • An Observational Study to Investigate Cognition and Quality of Life in Children and Adolescents with Dravet Syndrome (Pro00102865)
  • An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigation Studies of ISIS 396443
  • An open-label, long-term safety and tolerability study of b1ivaracetam as adjunctive treatment in pediatric study participants with epilepsy
  • Bloodwork_Clinical Outcomes Duchenne MD Therapeutic Trials-UCD0305
  • CINRG CHAR0312 Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
  • CINRG PITT0112 Becker Muscular Dystrophy-A Natural History Study to Predict Efficacy of Exon Skipping
  • CLN3: Modulation of Apoptosis and Ceramide Levels
  • Catastrophic outcomes in the AHC knock-in mouse models D801 N and E815K for ACH
  • Changes in Muscle Mass Using the D3-Creatine Dilution and Function in Neuromuscular Disease
  • Changes in muscle mass using the D3-creatine dilution method and function in DMD
  • Changes in muscle mass using the D3-creatine dilution method and function in DMD
  • Child Neurologist Career Development Program (CNCDP)
  • Clinical Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic Trials
  • Clinical Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic Trials
  • Consequences of prolonged febrile seizures in childhood
  • Continued anticonvulsants after resolution of neonatal seizures: a patient-centered comparative effectiveness study
  • Control Of Ionic Channels By Intracellular Calcium
  • Decision Aid for Infants with Neurology Conditions
  • Decision Making for Critically Ill Infants
  • Decision Making for Infants with Neurologic Conditions
  • Duchenne Outcomes Research Interchange Data Enrichment through EHR Extraction
  • E2007-G000-238 Perampanel for <2 yrs
  • Early Check CK Screening
  • Endogenous Opiods: Their Role In Ltp In Dentate Gyrus
  • Endogenous Opioids Regulate LTP of Inhibition
  • Endogenous Opioids Regulate Ltp Of Inhibition
  • Endogenous Opioids: Their Role In Ltp In Dentate Gyrus
  • Gaba-B Mediated Disinhibition In The Dentate Gyrus
  • Gabab Mediated Disinhibition In The Dentate Gyrus
  • Gabab Mediated Disinhibition In The Dentate Gyrus
  • Having the conversation: A communication guide for sudden unexpected death in epilepsy
  • Ion Channel Control By Calcium
  • La Marato de TV3 Foundation: TREAT-AHC, Identification of compounds for the treatment of Alternating Hemiplegia of Childhood by drug repositioning
  • LivaNova Core Registry
  • Long-Term, Open-Label Extension Study to Evaluate the Safety and Tolerability of NBI-827104 in Pediatric Subjects with Epileptic Encephalopathy with Continuous Spike-and-Wave During Sleep
  • Long-term Use of Viltolarsen in Boys with Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
  • Microburst VNS Therapy Feasibility Study in Subjects with Refractory Epilepsy
  • NSR-GENE (Neonatal Seizure Registry, GEnetics of post-Neonatal Epilepsy)
  • Neonatal Seizure Registry Developmental Functional EValuation (NSR-DEV)
  • Network Plasticity in Pediatric Traumatic Brain Injury- A Structural and Functional MRI Study of Network Plasticity in Pediatric Traumatic Brain Injury
  • NeuroSpeak: Developing a curriculum in delivering bad news for medical students in neurology
  • Neurotransmitter Modulation Of Intracellular Calcium
  • No question too small: Development of a question prompt list for infants and their families
  • OPEN-LABEL, SINGLE-ARM, MULTICENTER, LONG-TERM STUDY TO EVALUATE SAFETY AND EFFICACY OF BRIVARACETAM USED AS ADJUNCTIVE TREATMENT IN PEDIATRIC SUBJECTS WITH EPILEPSY
  • Open-Label Safety and Efficacy Study of Cenobamate (YKP3089) in Pediatric Subjects with Partial-onset (Focal) Seizures"
  • Open-label Comparison Study of Wearable Devices BioStamp, AIM Patch, and Actigraph as Compared to ActiMyo in User Preference and Device Performance for Ambulatory Duchenne's Muscular Dystrophy and Limb Girdle Muscular Dystrophy Patients
  • PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and Adults with Arginase 1 Deficiency
  • PPMD Research Liaison Grant
  • Pathophysiology of the AHC Knock-in Mouse Model
  • Pediatric Status Epilepticus Research Group
  • Ph I Zecuity
  • Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects with Epileptic Encephalopathy with Continuous Spike-and-Wav
  • Phase I, Open-Label Study of Carisbamate in Adult and Pediatric Subjects with Lennox-Gastaut Syndrome
  • Phase I, Open-Label, Pharmacokinetic, Dose Escalation Study of Carisbamate in Adult and Pediatric Subjects with Lennox-Gastaut Syndrome
  • Preventing Epilepsy Using Vigabatrin in Infants With Tuberous Sclerosis Complex (PREVeNT Trial)
  • SMA Care Center Registry
  • Spasm Prediction After SyMptomatic neonatal seizures - the SPASM study
  • Study of Oral Pyridoxal 5'-Phosphate for the Treatment of Patients with PNPO Deficiency
  • Using Longitudinal Data to Characterize the Natural History of Fragile X Syndrome - Component A
  • VBP-15-002 A Phase IIa Open-Label, Multiple Ascending Dose study to assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)
  • VBP15-003 A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)

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