The National Institute of Neurological Disease and Stroke,
The Office of Rare Diseases,*
The Johns Hopkins School of Medicine
and The PND Association Present
Medical Management of Pediatric Neurotransmitter Disease:
A Multidisciplinary Approach
Hyatt Dulles at Washington Dulles International Airport
Herndon, VA
Friday, July 18th - Saturday, July 19th 2008
Please click here for registration information.
Program Description & Objectives:
Although much progress has been made in the field of Pediatric Neurotransmitter Diseases (PND’s) much remains to be achieved in terms of diagnosis, understanding and treatment of these disorders. The conference will bring together scientists and clinicians to discuss current and potential treatments for PND’s and to arrive at practical guidelines for multidisciplinary treatment of the various PND’s.
The conference format features formal presentations by outstanding clinicians and researchers in the field, question and answer sessions, panel discussions and open dialogue. The conference format is designed to encourage interaction and collaboration between speakers and attendees concerning the best directions for clinical practice and research of PND’s. Families will have an opportunity to meet "face to face" with others dealing with the same disorders and the associated challenges.
After attending this conference participants will be able to:
-
Apply the latest scientific information toward the diagnosis and clinical management of PND’s as measured by earlier diagnosis, increased incidence and improved patient outcomes.
-
Recognize the rationale to consider and adopt a multidisciplinary treatment approach to PND’s as measured by enhanced patient outcomes after diagnosis.
-
Relate information gained to generate new research, new approaches and/or new collaborations on behalf of PND’s, as measured by increased research, new treatments and increased awareness.
Credit Designation Statement:
The Johns Hopkins University School of Medicine designates this educational activity for a maximum of 10.75 AMA PRA Category 1 Credit(s)™. Physicians should only claim credit commensurate with the extent of their participation in the activity.
Accreditation Statement:
This activity has been planned and implemented in accordance with the Essential Areas and policies of the Accreditation Council for Continuing Medical Education through the joint sponsorship of The Johns Hopkins University School of Medicine and The National Institutes of Health. The Johns Hopkins University School of Medicine is accredited by the ACCME to provide continuing medical education for physicians.
Who Should Attend:
This activity is intended for :
- Child Neurologists
- Metabolic Specialists
- Geneticists
- Health Care Professionals involved with neurological diseases
- Scientists
- Families and Caregivers
Event Sponsors:
Program Schedule **
Thursday July 17, 2008
7:00-8:30PM Welcome Reception
8:30-10:00PM PND Board of Direct and Medical & Scientific Advisory
Board Meeting
Friday July 18, 2008: Day 1
7:00-8:00AM Continental Breakfast/Registration
Morning Theme: Phenotypes and Therapy
Chair: Darryl De Vivo MD, Columbia, NY
8:00-8:15AM Welcome and Opening Remarks
Dan Tagle PhD, Program Director NIH/Neuroscience Center, Bethesda MD
Darryl DeVivo MD, Columbia, NY
8:15-9:00AM The Phenotypic Spectrum of Pediatric Neurotransmitter
Diseases and Infantile Parkinsomism
Roser Pons MD, Mitera, Greece
9:00-9:45PM Tetrahydrobiopterin dificiency in Pedatric
Neurotransmitter Disorders/Sepiaperin Reductase Deficiency
Nicola Longo MD, Primary Childrens Medical Center, UT
9:45-10:00AM Questions and Discussion
10:00-10:30AM Coffee/Break
10:30-11:15AM Treatment Approaches in - Hydroxybutyric Acidura:
Lessons from Mice and Men and the NIH protocols
Phillip Pearl MD, Children's National Wahsington, DC
11:15-12:00 AM Treatment Modalities for L-Aromatic Amino Acid
Decarboxylase Deficiency-The German Experience
Friederike Hoerster MD, University of Heidelberg, Germany
12:00-12:15PM Questions and Discussion
12:15-1:00PM Box Lunch
Afternoon Theme: Non-Pharmacological Intervention and Alternatives
Chair: K. Michael Gibson PhD FACMG,
Children's Hospital of Pittsburgh, PA
1:00-1:45PM Novel Treatment Approaches for Disorders of
Neurotransmission - Umbilical Stem Cells, Gene Therapy Update
Joanne Kurtzberg MD, Duke University, NC
Un Jung Kang MD, University of Chicago, IL
1:45-2:30PM Sensory Integration Intervention in PNDs
Sue Kratz OTR, CST, Special Therapies, WI
2:30-3:00PM Coffee/Break
3:00-3:45PM Physical and Occupational Intervention for Pediatric
Neurotransmitter Disorders-Goals and Objectives
Sally Evans MD, Children's National Washington, DC
3:45-4:30PM Deep Brain Stimulation as Adjuvant Therapy in
Neurotransmitter Disorders
Jerrold Vitek, MD PhD, Cleveland Clinic, OH
4:30-5:00PM Roundtable Discussion-Summary of Day
Darryl De Vivo MD, Columbia NY
5:00PM Adjourn
6:30PM Dinner
Saturday, July 19, 2008: Day 2
7:00-8:00AM Continental Breakfast
Morning Theme: Treatment Updates and Future Directions -
Short Presentations
Chair: Kathryn Swoboda MD FACMG, University of Utah
8:00-10:10AM Short presentations (20 min)
Topic Updates on Disorders of Neurotransmitter
8:00-8:20AM Sepiapterin Reductase Deficiency and the PND Database
Nicola Longo MD Salt Lake City, UT
8:20-8:40 AM Mouse Model Tyrosine Hydroxylase:
Clinical Variability in TH Deficiency as a Direct Consequence
of the Influence of the Genetic Mutation on the
Functions of the TH Enzyme
Ellen J. Hess PhD, Johns Hopkins Medicine, MD
8:40-9:00AM Questions and Discussion
9:00-9:30 AM Coffee/Break
9:30-9:50 AM Comprehensive Metabolite,
Enzyme and Molecular Diagnostics for PND’s
Keith Hyland PhD, Medical Neurogenetics LLC, GA
9:50-10:10AM Hepatocyte Repopulation in Gamma-Hydroxybutyric Aciduria
K. Michael Gibson PhD FACMG,
Children’s Hosptial of Pittsburgh, PA
10:10-10:30AM Combined Research/Clinical Fellowship for PND's
Michael Rotstein MD, Columbia, NY
10:30-10:45 AM Break
10:45-11:15 PM The Five Year Roadmap for the PND Association
PND Board of Directors
11:15AM-12:15PM Roundtable-questions and answers with all speakers
from both days.
12:15-12:30PM Closing Remarks
Darryl DeVivo MD/Dan Tagle PhD
Family Program Schedule
12:30 - 1:15PM Bag Lunch for families sponsored by PND Association
1:15-1:30PM Welcome/ Board Introductions, Nancy Speller President
Brad Hoffman Vice President/Treasurer
1:30-1:45PM Hope for Tomorrow Begins Today
PND Fundraising Committee
1:45-3:00PM Round Table Discussions Dopamine Related Diseases
and GABA Related Diseases
3:00-3:15PM Break
3:15-4:30PM Continued Round Table Discussions Dopamine
Related Diseases and GABA Related Diseases
4:30-4:45PM Closing Remarks/ Adjourn
*Funding for this conference was made possible (in part) by NS60363 from the National Institute of Neurological Disorders and Stroke (NINDS) and the Office of Rare Disorders (ORD). The views expressed in written conference materials or publications and by the speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services: nor does mention ofrade names, commercial practices or organizations imply endorsement bythe U.S. Government.
The Johns Hopkins University School of Medicine takes responsibility for the content, quality and scientific integrity of the CME activity.
**This agenda was prepared in October 2007 and is subject to change.
Session Chairs
Darryl C. De Vivo MD, Sidney Carter Professor of Neurology, Professor of Pediatrics, Director Pediatric Neurology Emeritus Columbia University Medical Center, New York NY
K. Michael Gibson PhD FACMG, Professor Pediatrics, Pathology & Human Genetics, Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine PA
Katherine Swoboda MD, FACMG, Associate Professor Neurology University of Utah School of Medicine, Salt Lake City UT
Dan Tagle PhD, Program Director NIH/Neuroscience Center, Bethesda MD
Presenters
Sally Evans MD, Division Chief Physical Medicine and Rehabilitation, Medical Director Rehabilitation, Children's National Medical Center
K. Michael Gibson PhD FACMG, Professor Pediatrics, Pathology & Human Genetics, Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, PA
Ellen J. Hess PhD, Associate Professor Neurology and Neuroscience, Johns Hopkins Medicine, MD
Friederike Hoerster MD, Department Child & Youth Medicine, University of Heidelberg, Germany
Keith Hyland PhD, Director, Department of Neurochemistry, Medical Neurogenetics LLC, GA
Un Jung Kang MD, Associate Professor of Neurology and Pharmacological and Physiological Sciences, Co Director Parkinson's Disease and Movement Disorders Center University of Chicago IL
Sue Kratz, OTR, CST, Director, Special Therapies, Milwaukee WI
Nicola Longo MD, PhD, Medical Genetics/Pediatric, Primary Childrens Medical Center, Salt Lake City UT
Joanne Kurtzburg MD, Chief Department Pediatrics Division of Blood and Marrow Transplantation, Duke University, Durham NC
Phillip Pearl MD, Associate Professor of Pediatrics and Neurology, Children's National Medical Center, Washington DC
Roser Pons MD, Pediatric Neurologist/Subspecialty in Movement Disorder, Hospital Agia Sofia, First Department of Pediatrics, University of Athens, Greece
Michael Rotstein MD, Clinical Fellowship, Columbia Presbyterian, NY
Jerrold Vitek, MD, PhD, Director Neuromodulation Research Center, Cleveland Clinic, OH
For further conference information please email
PND Operations Assistant, Stephanie Martin at [email protected]
Or call the PND Association at (516) 249-4948.
|