Rare Daily Staff

The CMT Research Foundation, which is focused on delivering treatments and cures for Charcot-Marie-Tooth disease, said it named additional scientific and clinical experts in Charcot-Marie-Tooth disease to its scientific advisory board.

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.

The scientific advisory board additions follow the unveiling of the not-for-profit organization. At that time, the organization named Richard Shimkets, president and CEO of Abeome, as the first member of the organization’s scientific advisory board.

The new members include:

  • Gabsang Lee, associate professor in the Department of Neurology and Institute for Cell Engineering at Johns Hopkins University
  • Thomas Lloyd, a physician-scientist at Johns Hopkins University specializing in hereditary motor neuron diseases
  • Alex Rossor, an honorary consultant neurologist and Wellcome Trust Post-Doctoral Clinical Fellow at the UCL Queen Square Institute of Neurology and the National Hospital for Neurology and Neurosurgery, London, United Kingdom

“This esteemed group brings a wealth of skills, knowledge and clinical expertise to the advisory board including use of innovative pathways like adult stem cells and gene therapy,” says Patrick Livney, CMTRF chairman of the board. “In particular, Dr. Lee’s expertise in the use of adult stem cells in drug screening brings the human element to a preclinical validation of candidate drugs and Dr. Shimkets’ work in biotech brings the industry voice to the vetting process.”

Livney said all four are leaders in pertinent industry groups like the Inherited Neuropathies Consortium, the Peripheral Nerve Society and The Antibody Society, giving them a holistic perspective of the research landscape for CMT.

The CMTRF said it expects to continue to add members to its scientific advisory board in the coming weeks to round out different aspects of drug development for CMT.


October 19, 2018
Photo: Richard Shimkets, CEO of Abeome