Article

VAL-1221 FOR THE TREATMENT OF PATIENTS WITH LAFORA DISEASE: STUDY PROTOCOL FOR A SINGLE-ARM, OPEN-LABEL CLINICAL TRIAL

2024-02-03

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Introduction</h4> Lafora Disease (LD) is an ultrarare fatal progressive myoclonic epilepsy, causing drug-resistant epilepsy, myoclonus, and psychomotor deterioration. LD is caused by mutations in EPM2A or NHLRC1, which lead to the accumulation of polyglucosans in the brain and neurodegeneration. There are no approved treatments for LD. VAL-1221 is a fusion protein comprised of the Fab portion...

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Literature Corpus work: a2fe30b5-0c2a-5c03-9595-24b08da310a2
DOI: 10.1101/2024.02.01.24302141

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VAL-1221 FOR THE TREATMENT OF PATIENTS WITH LAFORA DISEASE: STUDY PROTOCOL FOR A SINGLE-ARM, OPEN-LABEL CLINICAL TRIALDOI 10.1101/2024.02.01.24302141Read

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