PROGRAM SCHEDULE

Arrival and Registration

Welcoming Reception (private invitation only)  

  Saturday, May 18th 2002  

7:00

Registration and Breakfast  

8:30

Giovanna Spinella, MD -- 
Welcome and Opening Remarks  

8:45

Session I � Neurochemistry Associated
with Pediatric Neurotransmitter Diseases
Darryl C. De Vivo, MD -- Chair  

8:45

O.C. Snead, MD -- �Functional Aspects of GABA Neurotransmission�  

9:10

Teodoro Bottiglieri, PhD -- �Biogenic Amines-Metabolism and Regulation�  

9:40

O. C. Snead, MD -- �GHB: A Potential Neurotransmitter in Mammalian CNS�  

10:05

Roundtable Discussion  

10:20

Coffee Break  

10:35

Session II � Clinical Overview of PNDs
Andrea Gropman, MD -- Chair  

10:35

Kathryn Swoboda, MD -- �Aromatic L-Amino Acid Decarboxylase Deficiency�  

11:00

George F. Hoffmann, MD -- �Tyrosine Hydroxylase Deficiency�  

11:25

Masaya Segawa, MD -- �Hereditary Progressive Dystonia with marked Diurnal Fluctuation� (Dominantly Inherited GTP Cyclohydrolase 1 Deficiency)

11:50

Phillip Pearl, MD -- �Succinic Semialdehyde Dehydrogenase (SSADH) Deficiency (4-Hydroxybutyric Aciduria) in Children and Adults�  

12:15

Roundtable Discussion  

12:30

Lunch  

1:30

Session III --  Diagnoses and Therapeutic Intervention for PNDs
Keith Hyland, PhD -- Chair  

1:30

George F. Hoffmann, MD -- �The Clinician�s Approach to the Diagnosis of the Patient with a Suspected PND�  

Keith Hyland, PhD -- �The Lumbar Puncture for Diagnosis of PNDs�  

2:30

Roundtable Discussion  

2:45

Coffee Break  

3:00

Blair Ford, MD -- �Therapeutic Intervention for Disorders of Biogenic Amine Metabolism�  

3:30

Andrea Gropman, MD -- �Vigabatrin and Newer Interventions in SSADH Deficiency�  

3:55

Roundtable Discussion  

4:10

Summary Discussion -- �Strategy Planning for Implementation of Goals and Objectives of the PND Association�-Chaired by the Scientific Organization Committee and Invited Speakers (open to all participants)  

Adjourn  

Conference Dinner (private invitation only)  

Sunday, May 19, 2002

7:00

Breakfast  

8:15

Session IV � Animal Models of PNDs
Kathryn Swoboda, MD -- Chair  

8:15

Xiaoxi Zhuang, PhD -- �Animal Models of Catecholamine Defects�  

8:40

K. Michael Gibson, PhD -- �New Therapeutic Insights from a Murine Model of SSADH Deficiency�  

9:10

Keith Hyland, PhD -- �The hph-1 Mouse, a Model of GTP Cyclohydrolase Deficiency�  

9:35

Roundtable Discussion  

9:50

Coffee Break  

10:05

Session V --  Future Directions for Research And Treatment of PNDs
G. Hoffmann, MD -- Chair  

10:05

Jayanta Roy Chowdhury, MD -- �Liver Cell Transplantation and Liver Repopulation for Inherited Diseases�

10:35

Clive Svendsen, PhD �  �Genetic Modification of Human Neuronal Stem Cells; Implications for Brain  Repair�  

11:05

Roundtable Discussion  

11:15

Summary Discussion -- �What are the Future Objectives of the PND Association, and How Do We Coordinate to Achieve Our Goals�-Chaired by the Scientific Organizing Committee and Invited Speakers (open to all participants)  

12:10

Giovanna Spinella, MD and Darryl C. De Vivo, MD --Concluding Remarks and Closing  

Adjourn   

Program schedule is subject to change.   

If you have questions or if we can assist you in any way, 
please contact us at: (516) 937-0049 (phone and fax) or [email protected]

Hotel Information:  Springtime is a busy season in Washington D.C.  If you need a hotel room, please make reservations TODAY by contacting the Hyatt Regency Bethesda at (800) 233-1234.   When making reservations, mention the PND / NIH Medical Symposium.  Reduced rates have been negotiated for this conference.    

Registration Information:  To register for the PND / NIH Medical Symposium, Click on the Symposium Registration Form, fill in the requested information, print and mail the completed form with payment to the address at the bottom of the form.