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Treatment Options in Phenylketonuria and Related Pediatric Neurotransmitter Disorders
2 December 2008

Treatment Options in Phenylketonuria and Related Pediatric Neurotransmitter Disorders

The Trust is happy to support speakers for the ICIEM satellite meeting being held in San Diego on 29th August 2009, titled


"Treatment Options in Phenylketonuria and Related Pediatric Neurotransmitter Disorders"


We wish Professor Nenad Blau well with the symposium and feel sure that the awareness generated will go a long way towards assisting the attending Medical Professionals in their assessments to reach accurate diagnoses.  This will ultimately mean the correct subsequent treatment for the group of children with the still rare group of neurotransmitter diseases, of which AADC deficiency is one.



Visit http://www.biopku.org/sandiego2009/index.asp for full details and registration details.  Below is a copy of the symposium schedule:







Treatment Options in Phenylketonuria and Related Pediatric Neurotransmitter Disorders  


August 29, 2009 - San Diego - California




Symposium Schedule
Scientific Sessions


8:00 Registration Open


9:00-12:00 Morning session


Challenges in the pharmaceutical treatment of PKU; benefits and pitfalls


Chair: Barbara Burton


09:00-09:30     Assessing psychological benefits of new treatments for PKU - Susan Waisbren (USA)
09:30-10:00     Treatment of patients with PKU with BH4 - Alberto Burlina (Italy)
10:00-10:30     Combining BH4 and dietary therapy in patients with PKU - Rani H. Singh (USA)
                         Coffee
10:00-11:30
     The safety profile of BH4 in clinical practice - Amaya Bélanger-Quintana (Spain)
11:30-12:00     Pitfalls in determining BH4 responsiveness and in treating patients with BH4 - Barbara Burton (USA)


Lunch break


13:00—16:00 Afternoon session


Treatment of pediatric neurotransmitter disorders


Chair: Nenad Blau


13:00-13:30     Tyrosine hydroxylase deficiency - Georg Hoffmann (Germany)
13:30-14:00     Aromatic AA decarboxylase deficiency - Wang Tso Lee (Taiwan)
14:00-14:30     Tetrahydrobiopterin deficiencies - Nenad Blau (CH)
                         Coffee
15:00-15:30
     Dopa-responsive dystonia (GTPCH def.) - Kathy Swoboda (USA)
15:30-16:00     Sepiapterin reductase deficiency - Jennifer Friedman (USA)


15:00 – 17:00  ICIEM Registration Open


18:30               ICIEM Opening Welcome Reception



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