GNE Myopathy Disease Monitoring Program (GNEM-DMP) Steering Committee

The GNEM DMP was established by a team of researchers, doctors and patient representatives from USA, UK, Israel and Japan.  Representatives of the research team, clinicians, academics and patient representatives constitute the Steering Committee.  The Committee is overseeing the research and will ensure that the project is always acting in the patients best interests.

Members of the Steering Committee are:

Hanns Lochmüller Hanns Lochmüller - Chair of steering committee

Professor of Experimental Myology - University Medical Center Freiburg

Professor Hanns Lochmüller trained as a neurologist in Munich (Germany) and Montreal (Canada). He was appointed chair of experimental myology in the neuromuscular research group at the Institute of Genetic Medicine of Newcastle University in 2007. He was the first elected Chair of the TREAT-NMD Alliance Executive Committee.

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Zohar Argov Zohar Argov

Senior Academic Physician

Zohar Argov is a neurologist with a long-standing interest in muscle disorders. He first described the condition as Quadriceps-sparing or Inclusion body myopathy in Jewish patients which led to the identification of the causative gene. He is leading research programmes to develop better therapies for GNE myopathy ( and collaborates with Prof stella Mitrani-Rosenbaum's HIBM basic research laboratory.

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Emil D. Kakkis Emil D. Kakkis

Chief Executive Officer and President

Dr. Kakkis is best known for his work over the last 18 years to develop novel treatments for rare disorders. He began his work developing an enzyme replacement therapy (Aldurazyme®) for the rare disorder MPS I, with minimal funding and support. The struggle to get the therapy translated from a successful canine model to patients succeeded due to the critical financial support of the Ryan Foundation, a patient organization formed by Mark and Jeanne Dant for their son Ryan.

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Ichizo Nishino Ichizo Nishino

Director, Department of Neuromuscular Research - National Institute of Neuroscience - National Center of Neurology and Psychiatry

After having 5 years of clinical neurology training, Dr. Nishino started muscle disease research from 1994 under the tutelage of Dr. Ikuya Nonaka, who remained a steadfast source of inspiration. Later, his postdoctoral research for two years at Columbia University solidified his yearning to focus on research. In 2001, he was appointed directorship of the current Department of Neuromuscular Research and since then, he has been directing his team on the research to elucidate the pathomechanism and develop the therapy for hereditary muscle diseases, including DMRV/hIBM and various muscular dystrophies.


Oksana Pogoryelova Oksana Pogoryelova - Registry curator

Postdoctoral Research Associate - Newcastle University

Oksana completed medical training (MD) in Odessa (Ukraine) and PhD in clinical biochemistry in Aberdeen (UK). She has joined the Muscle team in Newcastle University (UK) in 2012 to work on the GNE myopathy project.

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Lalé Welsh Lalé Welsh

Executive Director, Neuromuscular Disease Foundation

Former Silicon Valley Executive (AT&T, Allianz, Monster), Lale Welsh has been a management consultant to nonprofits and governmental agencies, with the goal of applying compliant business solutions to the Public Benefit sector. Ms. Welsh is currently the Executive Director of The Neuromuscular DIsease Foundation in Beverly Hills, and works to promote research, funding and awareness for rare genetic diseases, and in particular, GNE myopathy (HIBM).

12 Apr 2017