<p>NMD are rare ion channel disorders. The optimal management and treatment of these disorders are not known. This multi-center proposal will allow the prospective collection of standardized data from a critical number of patients to help better define the clinical features of NDM. The data will also be used to establish clinically relevant endpoints for use in therapeutic trials. The identification and genetic characterization of patients will facilitate recruitment of participants for future therapeutic trials. Ultimately, the information gained will lead to the improvements in the treatment and management of NMD with an associated reduction in morbidity and improvement in patient quality of life.</p>

Nondystrophic Myotonias

2 selected publications including "Challenges in the design and conduct of therapeutic trials in channel ..." (8 citations)

Nondystrophic Myotonias

Challenges in the design and conduct of therapeutic trials in channel disorders

[Routine procedure or bubbles].