WO2013148740A1 - 4-aminopyridine à titre d'agent thérapeutique pour l'amyotrophie spinale (sma) - Google Patents

4-aminopyridine à titre d'agent thérapeutique pour l'amyotrophie spinale (sma) Download PDF

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Publication number
WO2013148740A1
WO2013148740A1 PCT/US2013/033952 US2013033952W WO2013148740A1 WO 2013148740 A1 WO2013148740 A1 WO 2013148740A1 US 2013033952 W US2013033952 W US 2013033952W WO 2013148740 A1 WO2013148740 A1 WO 2013148740A1
Authority
WO
WIPO (PCT)
Prior art keywords
smn
neurons
motor
mutants
sma
Prior art date
Application number
PCT/US2013/033952
Other languages
English (en)
Inventor
Brian Mccabe
Original Assignee
The Trustees Of Columbia University In The City Of New York
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by The Trustees Of Columbia University In The City Of New York filed Critical The Trustees Of Columbia University In The City Of New York
Priority to CN201380027537.0A priority Critical patent/CN104334174A/zh
Priority to EP13769101.0A priority patent/EP2830620A4/fr
Priority to JP2015503478A priority patent/JP2015512409A/ja
Priority to US14/388,421 priority patent/US20150064234A1/en
Publication of WO2013148740A1 publication Critical patent/WO2013148740A1/fr

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4409—Non condensed pyridines; Hydrogenated derivatives thereof only substituted in position 4, e.g. isoniazid, iproniazid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Abstract

On a découvert que l'inhibition pharmacologique des canaux K+ (à l'aide de l'antagoniste 4-AP à large spectre des canaux K+ approuvé par la FDA) favorisait les phénotypes de mutants smn, un résultat qui est cohérent avec l'excitabilité défectueuse des circuits moteurs par les influx de leurs interneurones et neurones sensoriels qui est une conséquence critique de la déplétion SMN. Sur la base de ces observations, certains modes de réalisation de l'invention concernent des méthodes de traitement de la SMA par administration de quantités thérapeutiquement efficaces d'un ou de plusieurs antagonistes des canaux potassiques, comprenant la 4-aminopyridine, la 4-(diméthylamino)pyridine, la 4-(méthylamino)pyridine, et la 4-(aminométhyl)pyridine. D'autres modes de réalisation concernent de nouvelles formulations pharmaceutiques comprenant deux antagonistes des canaux potassiques ou plus.
PCT/US2013/033952 2008-03-15 2013-03-26 4-aminopyridine à titre d'agent thérapeutique pour l'amyotrophie spinale (sma) WO2013148740A1 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CN201380027537.0A CN104334174A (zh) 2012-03-26 2013-03-26 作为脊髓性肌萎缩治疗剂的4-氨基吡啶
EP13769101.0A EP2830620A4 (fr) 2012-03-26 2013-03-26 4-aminopyridine à titre d'agent thérapeutique pour l'amyotrophie spinale (sma)
JP2015503478A JP2015512409A (ja) 2012-03-26 2013-03-26 脊髄性筋萎縮症用治療剤としての４−アミノピリジン
US14/388,421 US20150064234A1 (en) 2008-03-15 2013-03-26 4-aminopyridine as a therapeutic agent for spinal muscular atrophy

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261615466P 2012-03-26 2012-03-26
US61/615,466 2012-03-26

Publications (1)

Publication Number Publication Date
WO2013148740A1 true WO2013148740A1 (fr) 2013-10-03

Family

ID=49261189

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2013/033952 WO2013148740A1 (fr) 2008-03-15 2013-03-26 4-aminopyridine à titre d'agent thérapeutique pour l'amyotrophie spinale (sma)

Country Status (4)

Country Link
EP (1) EP2830620A4 (fr)
JP (1) JP2015512409A (fr)
CN (1) CN104334174A (fr)
WO (1) WO2013148740A1 (fr)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR3029113A1 (fr) * 2014-12-02 2016-06-03 Univ Paris-Sud Composes pour le traitement des maladies mitochondriales

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106511293B (zh) * 2015-09-11 2020-08-04 法德生技药品股份有限公司 含有达方吡啶的缓释口服制剂及其用途

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5952357A (en) * 1993-12-23 1999-09-14 Cornell Research Foundation, Inc. Treating diseases of the anterior horn cells
US20030060487A1 (en) * 2000-04-12 2003-03-27 Bamdad R. Shoshana Treatment of neurodegenerative disease
WO2011098449A1 (fr) * 2010-02-10 2011-08-18 Novartis Ag Procédés et composés pour la croissance de muscle
US20110207716A1 (en) * 2009-12-23 2011-08-25 Elan Pharmaceuticals, Inc. Inhibitors of polo-like kinase

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120010178A1 (en) * 2008-10-21 2012-01-12 President And Fellows Of Harvard College Methods and compounds for treatment of neurodegenerative disorders
JP5549260B2 (ja) * 2010-02-16 2014-07-16 富士通株式会社 ストレージシステムおよびデータ書込み方法
WO2012050884A2 (fr) * 2010-09-28 2012-04-19 President And Fellows Of Harvard College Glycosides cardiaques qui sont de puissants inhibiteurs de l'expression du gène de l'interféron bêta

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5952357A (en) * 1993-12-23 1999-09-14 Cornell Research Foundation, Inc. Treating diseases of the anterior horn cells
US20030060487A1 (en) * 2000-04-12 2003-03-27 Bamdad R. Shoshana Treatment of neurodegenerative disease
US20110207716A1 (en) * 2009-12-23 2011-08-25 Elan Pharmaceuticals, Inc. Inhibitors of polo-like kinase
WO2011098449A1 (fr) * 2010-02-10 2011-08-18 Novartis Ag Procédés et composés pour la croissance de muscle

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
See also references of EP2830620A4 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR3029113A1 (fr) * 2014-12-02 2016-06-03 Univ Paris-Sud Composes pour le traitement des maladies mitochondriales
WO2016087771A1 (fr) * 2014-12-02 2016-06-09 Universite Paris-Sud Composes pour le traitement des maladies mitochondriales
US10639287B2 (en) 2014-12-02 2020-05-05 Universite Paris-Sud Compounds for the treatment of mitochondrial diseases

Also Published As

Publication number Publication date
CN104334174A (zh) 2015-02-04
JP2015512409A (ja) 2015-04-27
EP2830620A4 (fr) 2015-12-09
EP2830620A1 (fr) 2015-02-04

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