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Important Updates in ADOA Research with Stoke Therapeutics


Stoke Therapeutics

The Massachusetts-based biotechnology company Stoke Therapeutics (Stoke) announced the enrollment of their first patient in a new study of people living with autosomal dominant optic atrophy (ADOA).


Stoke will be studying people between the ages of eight and 60 in the United States, United Kingdom, Italy, and Denmark with a clinical ADOA diagnosis. The study, called the FALCON Study, will follow patients for two years. During periodic assessments throughout the study period, researchers will evaluate each patient’s rate of vision impairment change and the progress of the disease.


Stoke is hoping to use data collected from the FALCON study in the preclinical development of a treatment for ADOA. Stoke is currently developing a proprietary, disease-modifying antisense oligonucleotide (ASO) called STK-002 to treat ADOA. Preclinical data demonstrated proof-of-mechanism and proof-of-concept results for STK-002, which earned Stoke’s product an orphan drug designation by the Federal Drug Administration (FDA).


There are no treatments or cures for ADOA currently available, and the FALCON study is a critical first step in the fight to treat ADOA. The study is expecting to enroll approximately 45 patients. The participants will be assessed at the beginning of their enrollment and subsequently six months, 12 months, 18 months, and 24 months after the baseline assessment.


The FALCON study is currently accepting patients. All questions about the study, and requests for enrollment information, can be sent to their email address: falconstudy@medpace.com.


At the ADOA Association, we’re constantly striving to keep a pulse on the important updates in ADOA research. Stay tuned in the coming months to learn more about future partnership opportunities ADOAA will be pursuing with Stoke Therapeutics.


Today, tomorrow and always we will continue the fight for sight. If you have questions about these research advancements, or want to share your family’s story with us, please don’t hesitate to reach out! Fill out the Contact Us form at the bottom of our website. And be sure to follow us on Facebook for frequent updates.


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