The Loire Valley Meeting (LVM) is a unique international scientific meeting taking place every two years in France.

As in previous years on behalf of INPDA, NPSuisse and Selbsthilfegruppe Germany are inviting researchers and clinicians from Europe and the US for discussing fundamental, translational, clinical and gene therapy research in an informal atmosphere with a view to outlining potential directions of future research activities. It is a restricted meeting.

Below are the videos of the presentations available for interested scientists, NP-C associations and NP-C families. All presentations are in English.




Session I:Fundamental ResearchChairman:
Jean Grünberg
Frank Pfrieger Exploring causes of neuronal vulnerability, new Biomarkers and new therapeutic targets in NPC1 disease - a progress report
Nick Platt Inflammation, Immune Responses and Therapy in NPC
Herman van der Putten Advances in research and therapy of juvenile (CLN3) Batten Disease
Stefan Kolb Repurposing an 'old' drug by modifying its properties - the case of fingolimod
Session II:
Clinical Research
Jim Green
Fran Platt NPC infectious disease side
Lola Ledesma Metabotropic Glutamate receptors in the pathology and treatment of NPC
Caroline Hasting 15 years from discovery to the beside: hydroxypropyl-beta-cyclodextrin. Where to now?
Elizabeth Berry-Kravis Long-Term Effects of Abrabetadex in NPC
Marc Patterson Small molecule therapies for NPC: miglustat, arimoclomol and N-acetyl-L-leucine
Session III:
Gene Therapy
Heiko Runz
Ahad Rahim The Development of Gene Therapy for Niemann-Pick C Disease
Cristin Davidson An update on murine gene therapy studies for Niemann-Pick type C1 disease
Fanny Collaud AAV vectors engineering for CNS-targeted gene therapy