Saturday, Sunday, Monday, Tuesday
Saturday July 14th, 2007
Joint NCL Congress and BDSRA session on Therapeutic approaches
1PM- 5PM
Welcoming remarks: David Pearce, University of Rochester Medical Center, USA
Session I: Therapeutic approaches for the NCLs
Chairs: David Pearce and Jane Emanuel
Plenary Lecture
Steven Goldman, University of Rochester Medical Center, USA
Stem Cells
NCL Plenary Lecture
Beverly Davidson, University of Iowa College of Medicine, USA
Approaching wide-spread enzyme replacement in patients with TPP-1 deficiency.
Invited Speakers
Robert Steiner, Oregon Health Science Center, USA
A Phase 1 Clinical Study of Human CNS Stem Cells (HuCNS-SC) in Patients with Neuronal Ceroid Lipofuscinosis
Ronald Crystal, Weill Cornell Medical College, USA
Adeno-associated Viral-mediated Gene Therapy for the CNS Manifestations of Batten Disease
Break
NCL presentations chosen from submitted abstracts.
Michael Chang, University of Iowa College of Medicine, USA - Intraventricular enzyme replacement in a mouse model of late infantile neuronal ceroid lipofuscinosis
Atilla Kovacs, University of Rochester Medical Center, USA - Effects of EGIS-8332, a non-competitive AMPA receptor antagonist, on the motor skills of Cln3-knockout mice
Laura Åberg, Helsinki University Central Hospital, Finland - Autoantibodies to GAD65 and intermittent prednisolone in juvenile neuronal ceroid lipofuscinosis
David Pearce, University of Rochester Medical Center, USA - Phenotypic amelioration of a behavioral deficit in Cln3-/- mice by immunosuppression
Krystyna Wisniewski, NYS Institute of Basic Research, USA - Clinical Trial: Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis with Late Infantile and Juvenile Onset
Open Discussion
Welcome reception and attendance of BDSRA Banquet