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Lafora Disease

Research & Development

We are planning to have more information here.

Current research

Valerion Therapeutics, a Massachusetts based biotechnology company is developing, VAL-0417, a novel fusion protein that combines Valerion’s delivery antibody (3E10) linked to amylase to uniquely target neuronal glycogen through enhanced delivery to affected tissues. In preclinical proof-of-concept studies, Valerion and collaborators at the University of Kentucky demonstrated in vitro and in vivo, the ability to deliver VAL-0417 to the skeletal muscle and brain with retention of up to 24 hours following intracerebroventricular (ICV) injection in Lafora mouse models.

These data were presented in a presentation titled “Lafora Body Degradation by a Therapeutic Enzyme for the Treatment of Lafora Disease,” at the 2017 Lafora Disease Workshop, a satellite symposium of the 32nd International Epilepsy Congress held September 2-6, 2017 in Barcelona, Spain.