Loire Valley Meeting 2022
The Loire Valley Meeting (LVM) was a unique international scientific meeting taking place every two years in France.
As in previous years on behalf of INPDA, NPSuisse and Selbsthilfegruppe Germany invited 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 was 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 | SPEAKER | TOPIC | VIMEO |
|---|---|---|---|
| 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 Chairman: 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 Chairman: 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 |