On September 2, 2016, BioPontis Alliance for Rare Diseases(http://biopontisalliance.org/), a unique international nonprofit organization, and VIB (http://www.vib.be/en/Pages/default.aspx), an excellence-based Life Science Research Institute in Belgium, announced a strategic partnership for rare diseases. The first program of the new partnership will be aimed at developing a treatment for one type of Charcot Marie Tooth disease (CMT), a rare, progressive, and invalidating neuropathy. There are no therapies available for CMT patients today, although it affects an estimated 1 in 2,500 people worldwide. The partnership will advance the work of one of VIB's premier researchers, Dr. Albena Jordanova at VIB-University of Antwerp. Patients with the form of CMT under investigation present symptoms common for many neuropathies and other neuromuscular diseases such as gradual weakness and wasting of the limbs, sensory loss, and inability to do daily tasks, but have an earlier onset of the disease. Lessons learned from the research into this specific form of CMT may hopefully help develop treatments for other forms of the disease. The partnership blends the basic research and early-stage translation resources at VIB with the professional therapeutics development expertise in the rare disease field at BioPontis Alliance. The public service missions of both partners enable the combined team to take bold steps in cracking the complex therapeutic challenges that a disease like CMT presents. By joining forces, both partners can step in earlier than most for-profit developers could justify. VIB and BioPontis Alliance will also collaborate to identify and jointly develop other VIB research projects with high promise to be developed into treatments.
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