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Items: 1 to 20 of 21

1.

Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.

Calder AN, Androphy EJ, Hodgetts KJ.

J Med Chem. 2016 Aug 16. [Epub ahead of print]

PMID:
27490705
2.

Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy.

Butchbach ME, Lumpkin CJ, Harris AW, Saieva L, Edwards JD, Workman E, Simard LR, Pellizzoni L, Burghes AH.

Exp Neurol. 2016 May;279:13-26. doi: 10.1016/j.expneurol.2016.02.009. Epub 2016 Feb 15.

PMID:
26892876
3.

Drug treatment for spinal muscular atrophy types II and III.

Wadman RI, Bosboom WM, van der Pol WL, van den Berg LH, Wokke JH, Iannaccone ST, Vrancken AF.

Cochrane Database Syst Rev. 2012 Apr 18;(4):CD006282. doi: 10.1002/14651858.CD006282.pub4. Review.

PMID:
22513940
4.

Drug treatment for spinal muscular atrophy types II and III.

Wadman RI, Bosboom WM, van den Berg LH, Wokke JH, Iannaccone ST, Vrancken AF.

Cochrane Database Syst Rev. 2011 Dec 7;(12):CD006282. doi: 10.1002/14651858.CD006282.pub3. Review. Update in: Cochrane Database Syst Rev. 2012;4:CD006282.

PMID:
22161400
5.

Treatment of spinal muscular atrophy cells with drugs that upregulate SMN expression reveals inter- and intra-patient variability.

Also-Rallo E, Alías L, Martínez-Hernández R, Caselles L, Barceló MJ, Baiget M, Bernal S, Tizzano EF.

Eur J Hum Genet. 2011 Oct;19(10):1059-65. doi: 10.1038/ejhg.2011.89. Epub 2011 May 25.

6.

LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate.

Garbes L, Riessland M, Hölker I, Heller R, Hauke J, Tränkle C, Coras R, Blümcke I, Hahnen E, Wirth B.

Hum Mol Genet. 2009 Oct 1;18(19):3645-58. doi: 10.1093/hmg/ddp313. Epub 2009 Jul 7.

7.

Drug treatment for spinal muscular atrophy types II and III.

Bosboom W, Vrancken AF, van den Berg LH, Wokke JH, Iannaccone ST.

Cochrane Database Syst Rev. 2009 Jan 21;(1):CD006282. doi: 10.1002/14651858.CD006282.pub2. Review. Update in: Cochrane Database Syst Rev. 2011;(12):CD006282.

PMID:
19160275
8.

Linking SMN to SMA: an assay for the rescuer.

Bönnemann CG, Finkel RS.

Neurology. 2008 Nov 25;71(22):1752-3. doi: 10.1212/01.wnl.0000335938.26633.e7. No abstract available.

PMID:
19029514
9.

Survival motor neuron gene 2 silencing by DNA methylation correlates with spinal muscular atrophy disease severity and can be bypassed by histone deacetylase inhibition.

Hauke J, Riessland M, Lunke S, Eyüpoglu IY, Blümcke I, El-Osta A, Wirth B, Hahnen E.

Hum Mol Genet. 2009 Jan 15;18(2):304-17. doi: 10.1093/hmg/ddn357. Epub 2008 Oct 29.

10.

A two-site ELISA can quantify upregulation of SMN protein by drugs for spinal muscular atrophy.

Nguyen thi Man, Humphrey E, Lam LT, Fuller HR, Lynch TA, Sewry CA, Goodwin PR, Mackenzie AE, Morris GE.

Neurology. 2008 Nov 25;71(22):1757-63. doi: 10.1212/01.wnl.0000313038.34337.b1. Epub 2008 Jul 16.

PMID:
18633133
11.

Spinal muscular atrophy.

Lunn MR, Wang CH.

Lancet. 2008 Jun 21;371(9630):2120-33. doi: 10.1016/S0140-6736(08)60921-6. Review.

PMID:
18572081
12.

A preliminary report on spinal muscular atrophy lymphoblastoid cell lines: are they an appropriate tool for drug screening?

Dayangaç-Erden D, Topaloğlu H, Erdem-Yurter H.

Adv Ther. 2008 Mar;25(3):274-9. doi: 10.1007/s12325-008-0030-1.

PMID:
18345520
13.

Clinical trials in spinal muscular atrophy.

Darras BT, Kang PB.

Curr Opin Pediatr. 2007 Dec;19(6):675-9. Review.

PMID:
18025935
14.

Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy.

Mercuri E, Bertini E, Messina S, Solari A, D'Amico A, Angelozzi C, Battini R, Berardinelli A, Boffi P, Bruno C, Cini C, Colitto F, Kinali M, Minetti C, Mongini T, Morandi L, Neri G, Orcesi S, Pane M, Pelliccioni M, Pini A, Tiziano FD, Villanova M, Vita G, Brahe C.

Neurology. 2007 Jan 2;68(1):51-5. Epub 2006 Nov 2.

PMID:
17082463
15.

Spinal muscular atrophy and therapeutic prospects.

Wirth B, Brichta L, Hahnen E.

Prog Mol Subcell Biol. 2006;44:109-32.

PMID:
17076267
16.

Spinal muscular atrophy: from gene to therapy.

Wirth B, Brichta L, Hahnen E.

Semin Pediatr Neurol. 2006 Jun;13(2):121-31. Review.

PMID:
17027862
17.

Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number.

Wirth B, Brichta L, Schrank B, Lochmüller H, Blick S, Baasner A, Heller R.

Hum Genet. 2006 May;119(4):422-8. Epub 2006 Mar 1.

PMID:
16508748
18.

New treatments for denervating diseases.

Pleasure D.

J Child Neurol. 2005 Mar;20(3):258-62.

PMID:
15832625
19.

Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients.

Brahe C, Vitali T, Tiziano FD, Angelozzi C, Pinto AM, Borgo F, Moscato U, Bertini E, Mercuri E, Neri G.

Eur J Hum Genet. 2005 Feb;13(2):256-9.

20.

Pilot trial of phenylbutyrate in spinal muscular atrophy.

Mercuri E, Bertini E, Messina S, Pelliccioni M, D'Amico A, Colitto F, Mirabella M, Tiziano FD, Vitali T, Angelozzi C, Kinali M, Main M, Brahe C.

Neuromuscul Disord. 2004 Feb;14(2):130-5.

PMID:
14733959
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