COMPOUNDS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY

Title:

COMPOUNDS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY

Document Type and Number:

WIPO Patent Application WO/2014/012050

Kind Code:

A3

Abstract:

Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.

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Inventors:

ANDROPHY ELLIOT J (US)
CHERRY JONATHON J (US)
CUNY GREGORY D (US)
GLICKSMAN MARCIE A (US)

Application Number:

PCT/US2013/050361

Publication Date:

March 20, 2014

Filing Date:

July 12, 2013

Export Citation:

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Assignee:

UNIV INDIANA RES & TECH CORP (US)
BRIGHAM & WOMENS HOSPITAL (US)
UNIV MASSACHUSETTS (US)

International Classes:

A61K31/42; C07D261/06; A61P21/00; C07D413/12

Domestic Patent References:

WO2007019417A1	2007-02-15
WO2009011850A2	2009-01-22
WO2009049180A2	2009-04-16
WO2009010454A2	2009-01-22

Foreign References:

US20060223873A1

2006-10-05

Other References: