Compositions and methods for the treatment of juvenile neuronal ceroid lipofuscinosis and related disorders

摘要

Provided are materials and methods for the prevention and treatment of Juvenile Neuronal Ceroid Lipofuscinosis comprising administration of an effective amount of at least one of a hemi-channel inhibitor or a phosphodiesterase-4 inhibitor. In some embodiments, the methods comprise administration of an effective amount of each of a hemi-channel inhibitor and a phosphodiesterase-4 inhibitor. Also provided are pharmaceutical compositions comprising a hemi-channel inhibitor or a phosphodiesterase-4 inhibitor, as well as kits comprising at least one effective dose of a hemi-channel inhibitor or a phosphodiesterase-4 inhibitor or a combination of both.

主权项

1. A method of reducing the rate of development of Juvenile Neuronal Ceroid Lipofuscinosis comprising administering an effective amount of a phosphodiesterase-4 inhibitor and an effective amount of INI-0602 to a subject.