Facio to present at World Orphan Drug Congress
Leiden, The Netherlands, November 3, 2017
Facio Therapies announced today that its managing director, David Dasberg, has been invited to speak at the 8th World Orphan Drug Congress (WODC), which will be held in Barcelona on 13-15 November 2017.
Undue production of DUX4 in skeletal muscle is the cause of FSHD. DUX4 sets in motion a cascade of biochemical events that eventually result in the devastating effects of FSHD. In people without FSHD, the production of DUX4 in skeletal muscle is repressed by regulatory mechanisms. Facio’s single goal is to develop a therapy that restores this repression as much as possible. In Barcelona, David will present Facio’s progress towards this goal.
FSHD (facioscapulohumeral dystrophy) is a skeletal muscle wasting disease that devastates the lives of about 700,000 people worldwide and those close to them. The loss of muscle strength has a huge impact on daily life. Living with FSHD means living with pain, fatigue, and social isolation. Above all, the future becomes uncertain because the course of the disease is unpredictable. About 20% of people with FSHD end up in a wheelchair. Currently, no therapy for FSHD is available other than forms of temporary symptomatic relief.
Facio Therapies, established in 2014, is a Netherlands-based company with a single focus – to overcome FSHD by developing a causal therapy that restores the natural repression of muscle-toxic DUX4. Facio works with leaders in the field to develop an affordable FSHD therapy in the most expeditious fashion possible. Rooted in, and dedicated to working for the FSHD community, Facio’s business approach is to have a positive impact on lives rather than to maximize financial gain. Since inception, Facio has raised over €8M in equity funding from FSHD-affected families, their friends, FSHD foundations, and Facio’s drug discovery partner, Evotec. Facio’s Board consists of business leaders from the FSHD community – Kees van der Graaf (Netherlands), Neil Camarta (Canada), Dave Mackay (USA), Bill Moss (Australia), and Chip Wilson (Canada) – and Evotec’s CSO, Cord Dohrmann.
The WODC is regarded as a key meeting in pushing forward with strategy, advocacy and partnering within the rare disease space. Speakers in Barcelona include representatives from rare disease companies, patient advocates, regulatory agencies, research institutes, and pharmaceutical companies.