EP 2830620 A1 20150204 - 4 AMINOPYRIDINE AS A THERAPEUTIC AGENT FOR SPINAL MUSCULAR ATROPHY
Title (en)
4 AMINOPYRIDINE AS A THERAPEUTIC AGENT FOR SPINAL MUSCULAR ATROPHY
Title (de)
4-AMINOPYRIDIN ALS THERAPEUTISCHES MITTEL FÜR SPINALE MUSKELATROPHIE
Title (fr)
4-AMINOPYRIDINE À TITRE D'AGENT THÉRAPEUTIQUE POUR L'AMYOTROPHIE SPINALE (SMA)
Publication
Application
Priority
- US 201261615466 P 20120326
- US 2013033952 W 20130326
Abstract (en)
[origin: WO2013148740A1] It has been discovered that pharmacological inhibition of K+ channels (using the FDA-approved broad-spectrum K+ channel antagonist 4-AP) positively benefitted smn mutant phenotypes, a result that is consistent with the defective excitability of motor circuits by their interneuron or sensory neuron inputs being a critical consequence of SMN depletion. Based on these observations, certain embodiments of the invention are directed to methods of treatment of SMA by administering therapeutically effective amounts of one or more potassium channel antagonists, including 4-aminopyridine, 4-(dimethylamino)pyridine, 4-(methylamino)pyridine, and 4-(aminomethyl)pyridine. Other embodiments are directed to new pharmaceutical formulations comprising two or more potassium channel antagonists.
IPC 8 full level
A61K 31/4409 (2006.01); A61P 21/00 (2006.01)
CPC (source: CN EP)
A61K 31/4409 (2013.01 - CN EP); A61P 21/00 (2017.12 - EP); A61P 21/02 (2017.12 - EP); A61P 43/00 (2017.12 - EP)
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
Designated extension state (EPC)
BA ME
DOCDB simple family (publication)
WO 2013148740 A1 20131003; CN 104334174 A 20150204; EP 2830620 A1 20150204; EP 2830620 A4 20151209; JP 2015512409 A 20150427
DOCDB simple family (application)
US 2013033952 W 20130326; CN 201380027537 A 20130326; EP 13769101 A 20130326; JP 2015503478 A 20130326