At Facio Therapies, we have a single focus - to find a cure for FSHD. More than 700,000 people worldwide are suffering from this progressive muscle wasting disease. Curing FSHD would require repairing certain genetic defects. That can't be done yet, but we do believe that we can tackle the cause of FSHD and stop the progression. Here's how we plan to take that important step.
The key event in FSHD is the unwanted production of a toxic protein called DUX4. We want to tackle the cause of this event.
The key event in FSHD is the unwanted production of a protein called DUX4. When produced in muscle tissue, DUX4 is highly toxic. In people without FSHD, the production of DUX4 is repressed. So this is what a causal FSHD therapy should do: restore that repression as much as possible. There is a natural mechanism that we aim to use for that purpose.
Research led by Drs Van der Maarel (Netherlands), Tapscott (US) and Tawil (US) has identified a natural repressor of DUX4: a protein called SMCHD1. In people with FSHD, SMCHD1 activity is too low. We will develop therapeutics that boost the activity of SMCHD1, such that DUX4 levels are strongly reduced. That way, we aim to stop the progression of FSHD.
Our therapy will be affordable and made available to as many people with FSHD as possible. We will develop our therapy as quickly and as cost-effectively as possible, using innovative ways to cut the duration and cost of drug development. Along the way, we will publish the results of every study we sponsor.
Our approach is different because we are for and by people with FSHD. We want to have a positive impact on lives rather than maximize financial gain. Our founders are three members of the FSHD community: business leaders Kees van der Graaf (Netherlands), Bill Moss (Australia), and Neil Camarta (Canada). With the support from people with FSHD, their families and their friends, we believe we will overcome FSHD together.
We are a network company working for the benefit of people with FSHD.Meet our team