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The clinical results of antisense oligonucleotide drugs to reduce the frequency of epileptic seizures in children by 70.6% are positive

 Last Update: 2022-01-11
 Source: Internet
 Author: User

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On December 3, 2021, Stoke Therapeutics announced that the ongoing Phase 1/2a clinical trial of STK-001 under investigation has achieved positive results

Dravet syndrome is usually associated with mutations in the SCN1A gene encoding the α subunit of the Nav1.

▲The mechanism of action of STK-001 (picture source: Stoke's official website)

The Phase 1/2a trial plans to enroll about 90 children and adolescents aged 2-18 years who are diagnosed with Dravet syndrome and have SCN1A gene mutations

STK-001 single dose (≤30 mg) and three doses (20 mg, once every 4 weeks) were well tolerated, and no safety issues related to the drug under investigation were reported

Note: The original text has been deleted

Reference materials:

[1] Stoke Therapeutics Presents Data from the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2021 Annual Meeting.

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