The first person in the world has received a dose of STK-001. Thank you to patient #1 and your family for your bravery and the trust you have placed in us.
#StokedAboutStoke
Stoke Therapeutics
779 posts
Stoke Therapeutics is addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
Joined February 2018
- Stoke announces positive new data from patients treated with STK-001 in ongoing Phase 1/2a MONARCH and ADMIRAL studies and the SWALLOWTAIL open-label extension study in children and adolescents with Dravet syndrome. Learn more here: investor.stoketherapeutics.com/news-releases/…
- It’s #BrainAwarenessWeek! We’re pleased to be collaborating with @AcadiaPharma to discover, develop & commercialize RNA-based medicines for the potential treatment of severe & rare genetic neurodevelopmental diseases of the CNS including #SYNGAP1 & #RettSyndrome.
00:00 - We are pleased to announce our plans to move forward with dosing of STK-001 in the MONARCH Phase 1/2a clinical study in children and adolescents with #Dravet syndrome. Read more in the press release:
- Tomorrow is #RareDiseaseDay. When we think about the #raredisease community, more people are impacted than we may realize. With a pipeline including 4 rare diseases, Dravet, ADOA, SYNGAP1, & Rett, we’re hopeful our work makes a difference for several rare conditions.
- #Dravet syndrome is a developmental & epileptic encephalopathy that causes developmental delays and cognitive impairment, in addition to seizures, due to a mutation in the SCN1A gene. Stoke aims to address root cause of Dravet, while current approaches only address seizures.
- Stoke received MHRA authorization to initiate a Phase 1/2a study of STK-001 for the treatment of #Dravetsyndrome in the U.K. The study complements ongoing U.S. studies & marks the first step in global expansion of clinical development of STK-001. For more: tinyurl.com/devyhevx
- #DYK the #SYNGAP1 gene is located on chromosome 6 on the loci 21.32 of the p arm? That’s why June 21 is SYNGAP1 Awareness Day. We are working with @Acadiapharma to develop & commercialize medicines for the potential treatment of SYNGAP1, a rare neurological disorder
00:00 - Today @AcadiaPharma and Stoke announced a collaboration to pursue multiple RNA-based treatments for severe and rare genetic neurodevelopmental diseases. Read the release for more info: investor.stoketherapeutics.com/news-releases/… #raredisease #JPM22
00:00 - Congratulations to @LoriLIsom1, Ph.D. for receiving the @AmEpilepsySoc Basic Science Research Award, which honors professional excellence in research for the improved understanding and treatment of #epilepsy.
- Congratulations to Stoke co-founder, director & @CSHL prof. Dr. Adrian Krainer on his election to @theNASciences for his distinguished work and commitment to advancing #RNAscience. #StokedAboutStoke nasonline.org/news-and-multi…
- CEO Edward Kaye presented at @cureSYNGAP1’s Conference and shared lessons learned from our work in #Dravetsyndrome. Stoke’s TANGO ASOs have the potential to improve treatment options for people with severe diseases, including SYNGAP1. #SRFConf22
- .@US_FDA has granted orphan drug designation for our lead product candidate, STK-001, an investigational new treatment for #Dravet syndrome, a severe and progressive genetic #epilepsy. ow.ly/GWI950vpSIb
- We had the opportunity to connect with families in the SYNGAP1 community at a local @cureSYNGAP1’s #Sprint4Syngap 5k in Lexington, Massachusetts. These events and personal stories help us learn about the disease and the importance of advancing our science.
















