DUX4-suppressing therapies are nearing the clinic

From our 2018 FSHD Connect conference, researchers present their work on therapies to suppress the toxic DUX4 gene. Two companies may be going into clinical trials in 2019. This is why it’s critically important to ramp up your support for our FSHD Therapeutics initiative, AND enroll in key studies such as the Clinical Trial Readiness study to ensure that these promising drugs have a pitfall-free path to FDA approval. 

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4 responses to “DUX4-suppressing therapies are nearing the clinic”

  1. My name is Jonas A. Minoza, 58 yrs old from Philippines. I was diagnosed with FSHD MD since I was 17. This kind of disease has lack of support froun our government and even private sectors. I would like to know if I could benefit this new drug.

    Thank you

    • It will still take some years for these promising candidates to go through the process of being tested in patients to see if they actually are effective (this is what we call a clinical trial), and then to be approved by the FDA. But what a change from a few years ago when there was nothing in the pipeline. The scientific basis for these drug candidates is also well-founded, so we are (cautiously) optimistic.

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