2022 International Congress » Therapy in Movement Disorders: Gene and Cell-Based Therapies

Meeting: 2022 International Congress

An AAV9 encoding human ABCD1 (SBT101) shows functional improvement following spinal cord delivery in a rodent model of adrenomyeloneuropathy

A. Pujol, A. Bosch, I. Dijkstra, S. Kemp, Y. Jaspers, S. Verdés, S. Fourcade, C. Guilera, M. Leal-Julià, A. Onieva, V. Vasireddy, SW. Clark, D. Anderson, K. Kozarsky (Barcelona, Spain)

Exosomes from human dental pulp stem cells as a cell-free drug delivery vehicle in vitro and in vivo chronic PD rat model through the intranasal route targeting efficacy & bio-distribution.

I. Datta, A. Kaushal, K. Mondal (Bangalore, India)

Fingolimod-responsive complex hyperkinetic movement disorder in neuromyelitis optica spectrum disorder

RE. Cortes, JA. Vatanagul, G. Saranza (Cebu, Philippines)

GDNF Gene Therapy for Parkinson’s Disease (PD): Preliminary Safety and Clinical Findings from a Phase Ib Study

A. van Laar, C. Christine, A. Merola, N. Phielipp, B. Elder, P. Larson, N. Stoicea, W. San Sebastian, M. Fiandaca, A. Kells, K. Bankiewicz (Research Triangle Park, USA)

Selection of clinical doses for SBT101, an AAV9-hABCD1 vector for the treatment of adrenomyeloneuropathy

D. Anderson, C. Maguire, C. Ng, Y. Gong, F. Eichler, S. Fourcase, C. Guilera, A. Pujol, A. Onieva, M. Leal-Julià, S. Verdés, A. Bosch, I. Dijkstra, S. Kemp, H. Park, T. Del Rio, T. Lutz, V. Vasireddy, S. Clark, K. Kozarsky (Bala Cynwyd, USA)

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