The Practice of Experimental Neurotherapeutics in Neuromuscular Disease

ABSTRACT PURPOSE OF REVIEW The discipline of experimental neurotherapeutics targets the process and operation of translating scientific discoveries into new treatments for neurologic diseases and has been instrumental in the progression of many areas of neurology. RECENT FINDINGS From the US Food and Drug Administration (FDA) market approval of the first systemic in vivo gene therapy in neurology to multiple current gene-targeting therapeutics, monoclonal antibodies, and new drugs under development or approved in the last several years, the field of experimental neurotherapeutics has a presence in every neuromuscular clinic in the United States. SUMMARY This article provides an overview of experimental neurotherapeutics with guidance on the clinical trials landscape, using examples in the field of neuromuscular disease. It covers the regulatory framework, clinical trial methodology, and offers advice on common pitfalls encountered when embarking on a clinical trials program in the clinic.