The National Institutes of Health and The PND Association Present

  Medical Management of Pediatric Neurotransmitter Disease:
A Multidisciplinary Approach

Friday July 20th-Saturday July 21st 2007

Please revisit this site for registration information.

Program  Description & Objectives

Although much progress has been made in the field of Pediatric Neurotransmitter Diseases much remains to be achieved in terms of diagnosis, understanding and treatment of these disorders. The conference will bring together scientists, clinicians and family members 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 will include formal presentations by outstanding clinicians and researchers in the field, question and answer sessions, panel discussions and open dialogue. Open discussions will be designed to encourage brainstorming between speakers and attendees about the best directions of clinical practice and research of pediatric neurotransmitter diseases. Families will have an opportunity to meet face to face with others dealing with the same disorders and challenges.

At the completion of this conference participants will:

• Be better prepared to apply the latest scientific information on PND's.
• Discuss the rationale and apply practical guidelines for a multidisciplinary treatment approach to PND's.
• Recognize the importance of sharing information amongst families, clinicans and scientists in the care and support of patients with PND's.
• Young investigators will increase their interest, collaboration, volume and depth of research, funding and publications in PND research.

Who Should Attend

Child neurologists, metabolic specialists, geneticists, health care professionals working with children with neurological disease, scientists, families and caregivers.

Accreditation 

This activity has been planned and implemented in accordance with the essential areas and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint sponsorship of The National Institutes of Health/Foundation for Advanced Education in the Sciences (NIH/FAES) and the PND Association. The NIH/FAES is accredited by the ACCME to provide continuing medical education for physicians.

Program Schedule 

Friday July 20, 2007:

Morning Theme:  Phenotypes and Therapy - Chair: Darryl De Vivo MD

7:00-8:00AM      Continental Breakfast

8:00-8:15AM      Welcome and Opening Remarks
                        Dan Tagle PhD, Darryl DeVivo MD

8:00-8:15AM      The phenotypic spectrum of Pediatric Neurotransmitter
                        Diseases and Infantile Parkinsomism, Marc Patterson MD

9:00-10:00AM     Novel treatment approaches for disorders of 
                        Neurotransmission - Umbilical Stem Cells, Gene Therapy Update
                        Joanne Kurtzberg MD, Un Jung Kang MD 

10:00-10:15AM Questions and Discussion

10:15-10:30AM  Coffee

10:30- 11:15AM  Treatment Approaches in -Hydroxybutyric Acidura: 
                        Lessons from mice and men and the NIH protocols
                        Phillip Pearl MD

11:15-12:00PM  The utility of tetrahydrobiopterin for intervention in Pedatric 
                       Neurotransmitter Disorders, Nenad Blau PhD 
                     
12:00-12:15PM  Questions and Discussion

12:15-1:30 PM   Box Lunch

Afternoon Theme: Non-Pharmacological Intervention and Alternatives - 
                                 Chair: Georg Hoffmann MD

1:30 -2:15PM  Nutritional considerations in the neurologically impaired 
                    child-relevance and application to neurotransmitter disorders
                    Nicole Walters RD

2:15- 3:00PM   Deep Brain Stimulation as adjuvant therapy in 
                     Neurotransmitter Disorders, Jonathan Mink MD PhD 

3:00-3:30PM    Coffee

3:30-4:15PM    Sensory Integration Intervention in PNDs, Sue Kratz OTR, CST

4:15-5:00PM    Physical and occupational intervention for Pediatric 
                     Neurotransmitter Disorders-Goals and Objectives
                     Sally Evans MD

5:00 -5:30PM   Roundtable Discussion-Summary of Day, Darryl De Vivo MD

5:30PM           Adjourn

7:00PM          Dinner 

July 21, 2007: Day 2
Morning Theme: Treatment Updates and Future Directions -
                              Short Presentations          
                              Chair: Kathryn Swoboda MD

8:00-8:20AM   Sepiapterin reductase deficiency and the Pediatric 
                     Neurotransmitter Disease Database, Nenad Blau PhD

8:20-8:40AM  Mutations Spectrum and Treatment Approaches in Defects 
                   of the Human Glycine Cleavage Complex in Patients
                   with NonKetotic Hyperglycinemia (NKH), Gunter Scharer MD

8:40-9:00AM  Questions and Discussion

9:00-9:30 AM  Coffee

9:30-9:50 AM Treatment modalities for L-aromatic amino acid decarboxylase 
                   deficiency-an International study group, Claudia Manegold MD

9:50-10:10 AM  Pyridoxine-Dependent Epilepsy:
                      How Difficult can it be to Diagnose?, Peter Clayton MD

10:10-10:30 AM  Coffee

10:30-11:00 PM   PND Board of Directors- The 5 year roadmap for the  PND
                        Association- accomplishments, goals and objectives, 
                        with introduction of new grantees/research fellow

11:00AM-12:00PM   Roundtable-questions and answers with all speakers
                           from both days.
  
12:00-12:15PM       Darryl DeVivo MD / Dan Tagle PhD-Closing Remarks

Family Program Schedule

12:15 - 1:00PM     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   

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
Georg F. Hoffman, MD Director Child and Youth Medicine, University of Heidelberg, Germany
Katherine Swoboda MD,  FACMG, Associate Professor Neurology University of Utah School of Medicine, Salt Lake City UT

Presenters
Nenad Blau PhD, Head, Laboratory for Tetrahydrobiopterin Research University of Children's Hospital, Zurich, Switzerland
Peter Clayton MD, Professor of Child Health & Pediatric Endocrinology University of Manchester Institute of Child Health, Manchester UK
Sally Evans MD, Division Chief Physical Medicine and Rehabilitation, Medical Director Rehabilitation, Children's National Medical Center
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
Sue Kratz, OTR, CST, Special Therapies, WI
Joanne Kurtzburg, MD, Chief Department Pediatrics Division of Blood and Marrow Transplantation, Duke University, Durham North Carolina
Claudia Manegold MD Department Child & Youth Medicine, University of Heidelberg, Germany
Jonathan Mink MD PhD Associate Professor of Neurology, Neurobiology & Anatomy and Pediatrics University of Rochester Medical Center, Rochester NY
Marc Patterson MD, Professor and Head, Divsion of Pediatric Neurology, Columbia University Medical Center, New York NY
Phillip Pearl MD, Associate Professor of Pediatrics and Neurology, Children's National Medical Center, Washington DC
Gunter Scharer MD, Assistant Professor of Pediatrics , University of Colorado at Denver & Health Science Center, Denver, CO
Nicole Waters RD Children's Hospital Pittsburgh, Pittsburgh PA
 

Medical & Scientific Symposia Committee
Chair- Darryl C. De Vivo MD, Sidney Carter Professor of Neurology, Professor of Pediatrics, Director Pediatric Neurology Emeritus Columbia University Medical Center, New York NY
Chair - K. Michael Gibson PhD FACMG, Professor Pediatrics, Pathology & Human Genetics, University Pittsburgh School of Medicine ,Children's Hospital Pittsburgh, Pittsburgh, PA
Nenad Blau PhD, Head, Laboratory for Tetrahydrobiopterin Research University of Children's Hospital, Zurich, Switzerland
Keith Hyland PhD, Director Department of Neurochemistry, Horizon Molecular Medicine, Atlanta GA
Georg F. Hoffman, MD Director Child and Youth Medicine, University of Heidelberg, Germany
Phillip Pearl MD, Associate Professor of Pediatrics and Neurology, Children's National Medical Center, Washington DC
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

PND Association Symposia Committee
Nancy Speller, President - Unknown Biogenic Amine Defect Rep
Brad Hoffman, VP/Treasurer -SSADH Rep
John Speller Assistant Treasurer, - Unknown Biogenic Amine Defect Rep
Kyler Drew, Secretary -TH Rep
Darlene Drew, Director-TH Rep
Carolyn Hoffman, Director -SSADH Rep
Julie Hoffman, Director -GTPCH Rep
Priya Kannusamy, Director -AADC Rep
Anne Kearney, SSADH Parent
Kimberly Speckman SSADH Parent
Liz McKinnon TH Parent
Stephanie Martin PND Staff -Operations Assistant