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For: Haddad H, Cifuentes-Diaz C, Miroglio A, Roblot N, Joshi V, Melki J. Riluzole attenuates spinal muscular atrophy disease progression in a mouse model. Muscle Nerve 2003;28:432-7. [PMID: 14506714 DOI: 10.1002/mus.10455] [Citation(s) in RCA: 64] [Impact Index Per Article: 2.9] [Reference Citation Analysis] [What about the content of this article? (0)] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/22/2022]
Number Cited by Other Article(s)
1
Moultrie F, Chiverton L, Hatami I, Lilien C, Servais L. Pushing the boundaries: future directions in the management of spinal muscular atrophy. Trends Mol Med 2025:S1471-4914(24)00339-3. [PMID: 39794178 DOI: 10.1016/j.molmed.2024.12.006] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 10/07/2024] [Revised: 12/10/2024] [Accepted: 12/11/2024] [Indexed: 01/13/2025]
2
Qiu J, Wu L, Qu R, Jiang T, Bai J, Sheng L, Feng P, Sun J. History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy. Front Cell Neurosci 2022;16:942976. [PMID: 36035257 PMCID: PMC9414009 DOI: 10.3389/fncel.2022.942976] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 05/13/2022] [Accepted: 07/20/2022] [Indexed: 11/21/2022]  Open
3
López-Cortés A, Echeverría-Garcés G, Ramos-Medina MJ. Molecular Pathogenesis and New Therapeutic Dimensions for Spinal Muscular Atrophy. BIOLOGY 2022;11:biology11060894. [PMID: 35741415 PMCID: PMC9219894 DOI: 10.3390/biology11060894] [Citation(s) in RCA: 5] [Impact Index Per Article: 1.7] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Academic Contribution Register] [Received: 04/05/2022] [Revised: 05/04/2022] [Accepted: 05/06/2022] [Indexed: 11/16/2022]
4
Lefebvre S, Sarret C. Pathogenesis and therapeutic targets in spinal muscular atrophy (SMA). Arch Pediatr 2021;27:7S3-7S8. [PMID: 33357595 DOI: 10.1016/s0929-693x(20)30269-4] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/15/2022]
5
Behera B. Nusinersen, an exon 7 inclusion drug for spinal muscular atrophy: A minireview. World J Meta-Anal 2021;9:277-285. [DOI: 10.13105/wjma.v9.i3.277] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Received: 12/03/2020] [Revised: 05/20/2021] [Accepted: 06/17/2021] [Indexed: 02/06/2023]  Open
6
In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy. Brain Sci 2021;11:brainsci11020194. [PMID: 33562482 PMCID: PMC7915832 DOI: 10.3390/brainsci11020194] [Citation(s) in RCA: 10] [Impact Index Per Article: 2.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 12/02/2020] [Revised: 01/29/2021] [Accepted: 02/01/2021] [Indexed: 12/19/2022]  Open
7
Menduti G, Rasà DM, Stanga S, Boido M. Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment. Front Pharmacol 2020;11:592234. [PMID: 33281605 PMCID: PMC7689316 DOI: 10.3389/fphar.2020.592234] [Citation(s) in RCA: 22] [Impact Index Per Article: 4.4] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 08/06/2020] [Accepted: 09/29/2020] [Indexed: 12/12/2022]  Open
8
New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand? Int J Mol Sci 2020;21:ijms21093297. [PMID: 32392694 PMCID: PMC7246502 DOI: 10.3390/ijms21093297] [Citation(s) in RCA: 76] [Impact Index Per Article: 15.2] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 04/12/2020] [Revised: 05/03/2020] [Accepted: 05/04/2020] [Indexed: 02/08/2023]  Open
9
Wadman RI, van der Pol WL, Bosboom WMJ, Asselman F, van den Berg LH, Iannaccone ST, Vrancken AFJE. Drug treatment for spinal muscular atrophy types II and III. Cochrane Database Syst Rev 2020;1:CD006282. [PMID: 32006461 PMCID: PMC6995983 DOI: 10.1002/14651858.cd006282.pub5] [Citation(s) in RCA: 16] [Impact Index Per Article: 3.2] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 12/22/2022]
10
Wadman RI, van der Pol WL, Bosboom WMJ, Asselman F, van den Berg LH, Iannaccone ST, Vrancken AFJE. Drug treatment for spinal muscular atrophy type I. Cochrane Database Syst Rev 2019;12:CD006281. [PMID: 31825542 PMCID: PMC6905354 DOI: 10.1002/14651858.cd006281.pub5] [Citation(s) in RCA: 6] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 12/12/2022]
11
Bozorg Qomi S, Asghari A, Salmaninejad A, Mojarrad M. Spinal Muscular Atrophy and Common Therapeutic Advances. Fetal Pediatr Pathol 2019;38:226-238. [PMID: 31060440 DOI: 10.1080/15513815.2018.1520374] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 10/26/2022]
12
Darras BT. Spinal muscular atrophies. Pediatr Clin North Am 2015;62:743-66. [PMID: 26022173 DOI: 10.1016/j.pcl.2015.03.010] [Citation(s) in RCA: 91] [Impact Index Per Article: 9.1] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 02/09/2023]
13
Kaczmarek A, Schneider S, Wirth B, Riessland M. Investigational therapies for the treatment of spinal muscular atrophy. Expert Opin Investig Drugs 2015;24:867-81. [DOI: 10.1517/13543784.2015.1038341] [Citation(s) in RCA: 19] [Impact Index Per Article: 1.9] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/21/2022]
14
Wynne GM, Russell AJ. Drug Discovery Approaches for Rare Neuromuscular Diseases. ORPHAN DRUGS AND RARE DISEASES 2014. [DOI: 10.1039/9781782624202-00257] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Subscribe] [Academic Contribution Register] [Indexed: 01/29/2023]
15
Genç B, Özdinler PH. Moving forward in clinical trials for ALS: motor neurons lead the way please. Drug Discov Today 2013;19:441-9. [PMID: 24171950 DOI: 10.1016/j.drudis.2013.10.014] [Citation(s) in RCA: 27] [Impact Index Per Article: 2.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 05/25/2013] [Revised: 09/07/2013] [Accepted: 10/21/2013] [Indexed: 12/12/2022]
16
Dachs E, Piedrafita L, Hereu M, Esquerda J, Calderó J. Chronic treatment with lithium does not improve neuromuscular phenotype in a mouse model of severe spinal muscular atrophy. Neuroscience 2013;250:417-33. [DOI: 10.1016/j.neuroscience.2013.07.026] [Citation(s) in RCA: 8] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 04/17/2013] [Revised: 06/26/2013] [Accepted: 07/11/2013] [Indexed: 12/12/2022]
17
Seo J, Howell MD, Singh NN, Singh RN. Spinal muscular atrophy: an update on therapeutic progress. Biochim Biophys Acta Mol Basis Dis 2013;1832:2180-90. [PMID: 23994186 DOI: 10.1016/j.bbadis.2013.08.005] [Citation(s) in RCA: 59] [Impact Index Per Article: 4.9] [Reference Citation Analysis] [Abstract] [Key Words] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 05/13/2013] [Revised: 07/27/2013] [Accepted: 08/14/2013] [Indexed: 12/24/2022]
18
The neuroprotective drug riluzole acts via small conductance Ca2+-activated K+ channels to ameliorate defects in spinal muscular atrophy models. J Neurosci 2013;33:6557-62. [PMID: 23575853 DOI: 10.1523/jneurosci.1536-12.2013] [Citation(s) in RCA: 42] [Impact Index Per Article: 3.5] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 11/21/2022]  Open
19
Therapeutic strategies for the treatment of spinal muscular atrophy. Future Med Chem 2013;4:1733-50. [PMID: 22924510 DOI: 10.4155/fmc.12.107] [Citation(s) in RCA: 18] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/13/2022]  Open
20
Viollet L, Melki J. Spinal muscular atrophies. HANDBOOK OF CLINICAL NEUROLOGY 2013;113:1395-411. [PMID: 23622363 DOI: 10.1016/b978-0-444-59565-2.00010-1] [Citation(s) in RCA: 6] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [Track Full Text] [Subscribe] [Academic Contribution Register] [Indexed: 12/25/2022]
21
Therapy development for spinal muscular atrophy in SMN independent targets. Neural Plast 2012;2012:456478. [PMID: 22701806 PMCID: PMC3369530 DOI: 10.1155/2012/456478] [Citation(s) in RCA: 17] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 03/01/2012] [Revised: 04/05/2012] [Accepted: 04/06/2012] [Indexed: 12/11/2022]  Open
22
Wadman RI, Bosboom WMJ, van der Pol WL, van den Berg LH, Wokke JHJ, Iannaccone ST, Vrancken AFJE. Drug treatment for spinal muscular atrophy types II and III. Cochrane Database Syst Rev 2012:CD006282. [PMID: 22513940 DOI: 10.1002/14651858.cd006282.pub4] [Citation(s) in RCA: 14] [Impact Index Per Article: 1.1] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 12/12/2022]
23
Wadman RI, Bosboom WMJ, van der Pol WL, van den Berg LH, Wokke JHJ, Iannaccone ST, Vrancken AFFJE. Drug treatment for spinal muscular atrophy type I. Cochrane Database Syst Rev 2012:CD006281. [PMID: 22513939 DOI: 10.1002/14651858.cd006281.pub4] [Citation(s) in RCA: 11] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 12/22/2022]
24
Spinal muscular atrophy: a clinical and research update. Pediatr Neurol 2012;46:1-12. [PMID: 22196485 DOI: 10.1016/j.pediatrneurol.2011.09.001] [Citation(s) in RCA: 116] [Impact Index Per Article: 8.9] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Received: 07/13/2011] [Accepted: 09/06/2011] [Indexed: 01/13/2023]
25
Wadman RI, Bosboom WM, van den Berg LH, Wokke JH, Iannaccone ST, Vrancken AF. Drug treatment for spinal muscular atrophy type I. Cochrane Database Syst Rev 2011:CD006281. [PMID: 22161399 DOI: 10.1002/14651858.cd006281.pub3] [Citation(s) in RCA: 6] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 01/13/2023]
26
Wadman RI, Bosboom WM, van den Berg LH, Wokke JH, Iannaccone ST, Vrancken AF. Drug treatment for spinal muscular atrophy types II and III. Cochrane Database Syst Rev 2011:CD006282. [PMID: 22161400 DOI: 10.1002/14651858.cd006282.pub3] [Citation(s) in RCA: 6] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 12/12/2022]
27
Spinal muscular atrophy: advances in research and consensus on care of patients. Curr Treat Options Neurol 2011;10:420-8. [PMID: 18990310 DOI: 10.1007/s11940-008-0044-7] [Citation(s) in RCA: 15] [Impact Index Per Article: 1.1] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 10/21/2022]
28
Burnett BG, Crawford TO, Sumner CJ. Emerging treatment options for spinal muscular atrophy. Curr Treat Options Neurol 2011;11:90-101. [PMID: 19210911 DOI: 10.1007/s11940-009-0012-x] [Citation(s) in RCA: 18] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/31/2022]
29
Abbara C, Estournet B, Lacomblez L, Lelièvre B, Ouslimani A, Lehmann B, Viollet L, Barois A, Diquet B. Riluzole pharmacokinetics in young patients with spinal muscular atrophy. Br J Clin Pharmacol 2011;71:403-10. [PMID: 21284699 DOI: 10.1111/j.1365-2125.2010.03843.x] [Citation(s) in RCA: 16] [Impact Index Per Article: 1.1] [Reference Citation Analysis] [Abstract] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/11/2022]  Open
30
Tiziano FD, Neri G, Brahe C. Biomarkers in rare disorders: the experience with spinal muscular atrophy. Int J Mol Sci 2010;12:24-38. [PMID: 21339974 PMCID: PMC3039940 DOI: 10.3390/ijms12010024] [Citation(s) in RCA: 12] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 11/10/2010] [Revised: 12/06/2010] [Accepted: 12/16/2010] [Indexed: 12/20/2022]  Open
31
Conserved genes act as modifiers of invertebrate SMN loss of function defects. PLoS Genet 2010;6:e1001172. [PMID: 21124729 PMCID: PMC2965752 DOI: 10.1371/journal.pgen.1001172] [Citation(s) in RCA: 77] [Impact Index Per Article: 5.1] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 02/04/2010] [Accepted: 09/21/2010] [Indexed: 01/27/2023]  Open
32
Treatment strategies for spinal muscular atrophy. Transl Neurosci 2010. [DOI: 10.2478/v10134-010-0045-4] [Citation(s) in RCA: 4] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 02/07/2023]  Open
33
Bosboom WMJ, Vrancken AFJE, van den Berg LH, Wokke JHJ, Iannaccone ST. Drug treatment for spinal muscular atrophy types II and III. Cochrane Database Syst Rev 2009:CD006282. [PMID: 19160275 DOI: 10.1002/14651858.cd006282.pub2] [Citation(s) in RCA: 8] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 12/12/2022]
34
Bosboom WMJ, Vrancken AFJE, van den Berg LH, Wokke JHJ, Iannaccone ST. Drug treatment for spinal muscular atrophy type I. Cochrane Database Syst Rev 2009:CD006281. [PMID: 19160274 DOI: 10.1002/14651858.cd006281.pub2] [Citation(s) in RCA: 8] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 01/16/2023]
35
Vitte J, Attali R, Warwar N, Gurt I, Melki J. Spinal muscular atrophy. ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY 2009;652:237-46. [PMID: 20225030 DOI: 10.1007/978-90-481-2813-6_16] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.2] [Reference Citation Analysis] [Abstract] [Track Full Text] [Subscribe] [Academic Contribution Register] [Indexed: 12/20/2022]
36
Pane M, Staccioli S, Messina S, D'Amico A, Pelliccioni M, Mazzone ES, Cuttini M, Alfieri P, Battini R, Main M, Muntoni F, Bertini E, Villanova M, Mercuri E. Daily salbutamol in young patients with SMA type II. Neuromuscul Disord 2008;18:536-40. [PMID: 18579379 DOI: 10.1016/j.nmd.2008.05.004] [Citation(s) in RCA: 75] [Impact Index Per Article: 4.4] [Reference Citation Analysis] [Abstract] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 03/01/2008] [Revised: 05/05/2008] [Accepted: 05/12/2008] [Indexed: 11/15/2022]
37
Lunn MR, Wang CH. Spinal muscular atrophy. Lancet 2008;371:2120-33. [PMID: 18572081 DOI: 10.1016/s0140-6736(08)60921-6] [Citation(s) in RCA: 605] [Impact Index Per Article: 35.6] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 01/19/2023]
38
Swoboda KJ, Kissel JT, Crawford TO, Bromberg MB, Acsadi G, D'Anjou G, Krosschell KJ, Reyna SP, Schroth MK, Scott CB, Simard LR. Perspectives on clinical trials in spinal muscular atrophy. J Child Neurol 2007;22:957-66. [PMID: 17761650 PMCID: PMC3260051 DOI: 10.1177/0883073807305665] [Citation(s) in RCA: 44] [Impact Index Per Article: 2.4] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 01/02/2023]
39
Wirth PhD B, Riessland Msc M, Hahnen MBA E. Drug discovery for spinal muscular atrophy. Expert Opin Drug Discov 2007;2:437-51. [DOI: 10.1517/17460441.2.4.437] [Citation(s) in RCA: 4] [Impact Index Per Article: 0.2] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/18/2022]
40
Avila AM, Burnett BG, Taye AA, Gabanella F, Knight MA, Hartenstein P, Cizman Z, Di Prospero NA, Pellizzoni L, Fischbeck KH, Sumner CJ. Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy. J Clin Invest 2007;117:659-71. [PMID: 17318264 PMCID: PMC1797603 DOI: 10.1172/jci29562] [Citation(s) in RCA: 268] [Impact Index Per Article: 14.9] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 06/30/2006] [Accepted: 12/20/2006] [Indexed: 01/20/2023]  Open
41
Spinal Muscular Atrophy. Neurobiol Dis 2007. [DOI: 10.1016/b978-012088592-3/50046-3] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.1] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 11/19/2022]  Open
42
Tsai LK, Tsai MS, Lin TB, Hwu WL, Li H. Establishing a standardized therapeutic testing protocol for spinal muscular atrophy. Neurobiol Dis 2006;24:286-95. [PMID: 16952456 DOI: 10.1016/j.nbd.2006.07.004] [Citation(s) in RCA: 46] [Impact Index Per Article: 2.4] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/30/2006] [Revised: 07/04/2006] [Accepted: 07/10/2006] [Indexed: 01/19/2023]  Open
43
Mattis VB, Rai R, Wang J, Chang CWT, Coady T, Lorson CL. Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts. Hum Genet 2006;120:589-601. [PMID: 16951947 DOI: 10.1007/s00439-006-0245-7] [Citation(s) in RCA: 78] [Impact Index Per Article: 4.1] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 07/07/2006] [Accepted: 08/10/2006] [Indexed: 11/24/2022]
44
Wishart TM, Parson SH, Gillingwater TH. Synaptic Vulnerability in Neurodegenerative Disease. J Neuropathol Exp Neurol 2006;65:733-9. [PMID: 16896307 DOI: 10.1097/01.jnen.0000228202.35163.c4] [Citation(s) in RCA: 149] [Impact Index Per Article: 7.8] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 10/22/2022]  Open
45
Wirth B, Brichta L, Hahnen E. Spinal muscular atrophy: from gene to therapy. Semin Pediatr Neurol 2006;13:121-31. [PMID: 17027862 DOI: 10.1016/j.spen.2006.06.008] [Citation(s) in RCA: 92] [Impact Index Per Article: 4.8] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 11/23/2022]
46
Nelson L, Owens H, Hynan LS, Iannaccone ST. The gross motor function measure™ is a valid and sensitive outcome measure for spinal muscular atrophy. Neuromuscul Disord 2006;16:374-80. [PMID: 16632361 DOI: 10.1016/j.nmd.2006.03.005] [Citation(s) in RCA: 61] [Impact Index Per Article: 3.2] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 08/07/2005] [Revised: 12/17/2005] [Accepted: 03/07/2006] [Indexed: 11/17/2022]
47
Sumner CJ. Therapeutics development for spinal muscular atrophy. NeuroRx 2006;3:235-45. [PMID: 16554261 PMCID: PMC3593434 DOI: 10.1016/j.nurx.2006.01.010] [Citation(s) in RCA: 68] [Impact Index Per Article: 3.6] [Reference Citation Analysis] [Abstract] [MESH Headings] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 01/09/2023]
48
Baughan T, Shababi M, Coady TH, Dickson AM, Tullis GE, Lorson CL. Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. Mol Ther 2006;14:54-62. [PMID: 16580882 DOI: 10.1016/j.ymthe.2006.01.012] [Citation(s) in RCA: 58] [Impact Index Per Article: 3.1] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 12/21/2005] [Revised: 01/26/2006] [Accepted: 01/31/2006] [Indexed: 11/29/2022]  Open
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