MONARCH Study Update

By |2020-08-17T14:48:44-05:00August 18th, 2020|Categories: Research / Treatments|

First patient dosed with STK-001 in Part A of Phase 1/2a MONARCH clinical trial Stoke Therapeutics recently began the Monarch clinical trial and just announced that they have successfully administered STK-001 to the first patient in their trial. STK-001 is a novel disease-modifying therapy that utilizes RNA antisense oligonucleotides (ASOs) to restore levels [...]

Day of Dravet Virtual Workshop – October 17, 2020

By |2020-08-13T17:39:42-05:00August 14th, 2020|Categories: Decoding Dravet Blog|

Each fall, the Dravet community looks forward to DSF’s Day of Dravet regional workshops. As a part of our Family Network program, DSF usually hosts five workshops each fall across the U.S. to bring our community together in person to learn about the latest in Dravet syndrome, connect with one another, and be a part [...]

Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression

By |2020-09-04T01:54:18-05:00August 13th, 2020|Categories: DSF Research Review|Tags: , , , |

Stoke Therapeutics published a paper in Nature Communications detailing the mechanism of action for their new TANGO (targeted augmentation of nuclear gene output) therapy that utilizes ASO (antisense oligonucleotide) technology. While the applications of this technology have been discussed in presentations and meetings, this is the first publication detailing how it was developed. Cells use [...]

Back to School Anxiety in the Dravet Community

By |2020-08-06T09:31:28-05:00August 7th, 2020|Categories: Decoding Dravet Blog|

While the pandemic has had a devastating effect for all students, most special ed students have had a particularly difficult time adapting to distance learning. They miss their routine, their therapies, and the opportunity for socialization. Parents who have had to step in as “teacher and therapist” during distance learning often report that they are [...]

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