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Overcoming Hurdles


overcoming Hurdles

Consistent cost management will enable us to maximize therapy access by making it affordable and reimbursable. That way, we are able to pursue a socially responsible business model that creates value for patients as well as shareholders.

With our single focus on fshd, we devote our capital to doing only what is needed to translate recent scientific advancements into a causal therapy. By clicking continue below and using our sites or applications, you agree that we and our third party advertisers can: transfer your personal data to the United States or other countries, and process your personal data to serve you with personalized ads, subject to your. Our founders are three members of the fshd community: business leaders Kees van der Graaf (Netherlands Bill Moss AO (Australia and Neil Camarta (Canada). In fact, we screened over 34,000 small molecule compounds, and obtained over 300 so-called hits and identified novel approaches to repress the production of the muscle-toxic DUX4 protein. You always have the choice to experience our sites without personalized advertising based on your web browsing activity by visiting the. In addition, we are preparing for the development of lead compounds for preclinical and clinical development. We are funded by fshd-affected families and friends from Australia, Europe, and North America, by the fshd Global Research Foundation (Australia the (Dutch) fshd Stichting, the fshd Canada Foundation, and Amis FSH (France and by our drug discovery partner, Evotec (Germany).

Discovering effective ways to repress and prevent the production of DUX4 is extremely challenging because of DUX4s naturally elusive behavior and the many technical hurdles for its detection in muscle. Even if you choose not interment of Japanese American to have your activity tracked by third parties for advertising services, you will still see non-personalized ads on our site. Foaled in 1995, breed: Thoroughbred, foaled in 1988, breed: Quarter Horse. When produced in muscle tissue, DUX4 is highly toxic due to a cascade of events eventually resulting in the devastating effects of fshd. Also, we will reinvest part of our future profits into projects to expand quality-of-life options. By overcoming this challenge, we have created an essential platform for the discovery of innovative approaches to tackle the cause of fshd. Foaled in 2003, breed: Appendix Quarter Horse, read More Back to top. Unfortunately, our website is currently unavailable in most European countries. This way, we are working towards a much needed treatment for fshd, without cutting corners. However, these are no medicines for fshd yet. In people without fshd, the production of DUX4 is repressed by regulatory mechanisms in the muscle cell.

overcoming Hurdles


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