There are now plans in place to study a gene therapy for GNE myopathy (GNEM), with human dosing expected to begin in 24 months. The plans were developed by the Neuromuscular Disease Foundation (NDF), one of the global leaders in GNEM research and treatment development.

Human Dosing

In order to reach the human dosing phase of a clinical trial, the NDF has made two changes to its drug development process. The first change was to include two brilliant, world-renowned scientists: Dr. Angela Lek and Rich Horgan. Both are experts in neuromuscular diseases and can bring extremely helpful skillsets.

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