Not-for-profit Launched to Drive Treatments and Cures for Charcot-Marie-Tooth

Rare Daily Staff

Two leading Charcot-Marie-Tooth patient-advocates have launched the CMT Research Foundation, a not-for-profit organization focused on delivering treatments and cures for CMT.

CMT is a group of progressive genetic nerve diseases and can lead to severe disability or even death. There are more than 90 known genetic causes for the condition. Onset can be at birth or later in life and is characterized by loss of muscles and sensation in the feet, hands, legs, and arms. Currently there is no cure for CMT and there are no effective treatments.

Patrick Livney, a former chairman of the Charcot-Marie-Tooth Association, and Susan Ruediger, a former Charcot-Marie-Tooth Association development director, founded the CMT Research Foundation. Livney is serving as chairman and Ruediger is the new organization’s CEO.

The foundation said it will support the most promising and effective research by developing strategic alliances with academic and industry stakeholders. With the help of an advisory board comprised of academic, medical, and biotech experts, the foundation will fund translational research for all types of CMT.

“Research is what it takes to develop effective treatments and ultimately a cure for CMT and the only thing standing in the way of that research is funding,” said Ruediger. “As a patient living with CMT every day, I know firsthand how urgent it is that we eliminate this devastating condition and I won’t stop until there is a cure.”

The research foundation’s board of directors and scientific advisory board are still in formation. In addition to Liveney and Ruediger, its board of directors includes:

• Paul August, vice-president, biology, ICAGEN
• Adam Kauffman, a CMT patient and software engineer specializing in internet security
• Michelle Moon, a neurologist who lost her daughter to CMT
• Cleary Simpson, an experienced media executive whose daughter has CMT.

Richard Shimkets, president and CEO of Abeome, is the first member of the organization’s scientific advisory board.

September 19, 2018
Photo: Susan Ruediger and Patrick Livney, Co-founders of the Charcot-Marie-Tooth Association