US 12,083,105 B2
Compositions and methods for treating adult-onset neuronal ceroid lipofuscinosis (Kufs disease)
Manu Sharma, Tappan, NY (US); and Nima N. Naseri, Philadelphia, PA (US)
Assigned to Cornell University, Ithaca, NY (US)
Filed by CORNELL UNIVERSITY, Ithaca, NY (US)
Filed on Jan. 22, 2021, as Appl. No. 17/155,200.
Claims priority of provisional application 62/993,131, filed on Mar. 23, 2020.
Prior Publication US 2021/0290602 A1, Sep. 23, 2021
Int. Cl. A61K 31/44 (2006.01); A61K 9/00 (2006.01); A61K 31/16 (2006.01); A61K 45/06 (2006.01); A61P 25/28 (2006.01)
CPC A61K 31/44 (2013.01) [A61K 9/0014 (2013.01); A61K 9/0019 (2013.01); A61K 9/0053 (2013.01); A61K 9/0073 (2013.01); A61K 31/16 (2013.01); A61K 45/06 (2013.01); A61P 25/28 (2018.01)] 10 Claims
OG exemplary drawing
 
1. A method for preventing or treating Kufs Disease in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of an iron chelator, wherein the subject harbors a dominant negative CSPα mutation.