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For: Calder AN, Androphy EJ, Hodgetts KJ. Small Molecules in Development for the Treatment of Spinal Muscular Atrophy. J Med Chem 2016;59:10067-10083. [PMID: 27490705 PMCID: PMC5744254 DOI: 10.1021/acs.jmedchem.6b00670] [Citation(s) in RCA: 46] [Impact Index Per Article: 5.1] [Reference Citation Analysis] [What about the content of this article? (0)] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/11/2022]
Number Cited by Other Article(s)
1
Basak S, Biswas N, Gill J, Ashili S. Spinal Muscular Atrophy: Current Medications and Re-purposed Drugs. Cell Mol Neurobiol 2024;44:75. [PMID: 39514016 PMCID: PMC11549153 DOI: 10.1007/s10571-024-01511-3] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 07/31/2024] [Accepted: 10/30/2024] [Indexed: 11/16/2024]
2
Haque US, Yokota T. Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges. Genes (Basel) 2024;15:999. [PMID: 39202360 PMCID: PMC11353366 DOI: 10.3390/genes15080999] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 06/28/2024] [Revised: 07/20/2024] [Accepted: 07/22/2024] [Indexed: 09/03/2024]  Open
3
Bagga P, Singh S, Ram G, Kapil S, Singh A. Diving into progress: a review on current therapeutic advancements in spinal muscular atrophy. Front Neurol 2024;15:1368658. [PMID: 38854961 PMCID: PMC11157111 DOI: 10.3389/fneur.2024.1368658] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/11/2024] [Accepted: 04/29/2024] [Indexed: 06/11/2024]  Open
4
Kolić D, Šinko G, Jean L, Chioua M, Dias J, Marco-Contelles J, Kovarik Z. Cholesterol Oxime Olesoxime Assessed as a Potential Ligand of Human Cholinesterases. Biomolecules 2024;14:588. [PMID: 38785995 PMCID: PMC11117805 DOI: 10.3390/biom14050588] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 04/08/2024] [Revised: 05/10/2024] [Accepted: 05/14/2024] [Indexed: 05/25/2024]  Open
5
Ponomarev AS, Chulpanova DS, Yanygina LM, Solovyeva VV, Rizvanov AA. Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape. Int J Mol Sci 2023;24:13743. [PMID: 37762045 PMCID: PMC10530942 DOI: 10.3390/ijms241813743] [Citation(s) in RCA: 6] [Impact Index Per Article: 3.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 06/30/2023] [Revised: 08/28/2023] [Accepted: 08/29/2023] [Indexed: 09/29/2023]  Open
6
Li Q, Kang C. Targeting RNA-binding proteins with small molecules: Perspectives, pitfalls and bifunctional molecules. FEBS Lett 2023;597:2031-2047. [PMID: 37519019 DOI: 10.1002/1873-3468.14710] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 03/01/2023] [Revised: 06/26/2023] [Accepted: 06/28/2023] [Indexed: 08/01/2023]
7
Meanwell NA. The pyridazine heterocycle in molecular recognition and drug discovery. Med Chem Res 2023;32:1-69. [PMID: 37362319 PMCID: PMC10015555 DOI: 10.1007/s00044-023-03035-9] [Citation(s) in RCA: 10] [Impact Index Per Article: 5.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/07/2023] [Accepted: 02/06/2023] [Indexed: 03/17/2023]
8
Sabuncu Gürses G, Erdem SS, Saçan MT. A QSAR study to predict the survival motor neuron promoter activity of candidate diaminoquinazoline derivatives for the potential treatment of spinal muscular atrophy. SAR AND QSAR IN ENVIRONMENTAL RESEARCH 2023;34:247-266. [PMID: 37125536 DOI: 10.1080/1062936x.2023.2200975] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Academic Contribution Register] [Indexed: 05/03/2023]
9
Lejman J, Panuciak K, Nowicka E, Mastalerczyk A, Wojciechowska K, Lejman M. Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives. Int J Mol Sci 2023;24:ijms24021130. [PMID: 36674643 PMCID: PMC9860634 DOI: 10.3390/ijms24021130] [Citation(s) in RCA: 9] [Impact Index Per Article: 4.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 11/28/2022] [Revised: 12/31/2022] [Accepted: 01/04/2023] [Indexed: 01/11/2023]  Open
10
Morelli C, Ingrasciotta G, Jacoby D, Masri A, Olivotto I. Sarcomere protein modulation: The new frontier in cardiovascular medicine and beyond. Eur J Intern Med 2022;102:1-7. [PMID: 35534374 DOI: 10.1016/j.ejim.2022.04.020] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Received: 02/15/2022] [Revised: 04/14/2022] [Accepted: 04/17/2022] [Indexed: 01/10/2023]
11
Kanda S, Moulton E, Butchbach MER. Effects of Inhibitors of SLC9A-Type Sodium-Proton Exchangers on Survival Motor Neuron 2 (SMN2) mRNA Splicing and Expression. Mol Pharmacol 2022;102:92-105. [PMID: 35667685 PMCID: PMC9341265 DOI: 10.1124/molpharm.122.000529] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 03/15/2022] [Accepted: 05/09/2022] [Indexed: 11/22/2022]  Open
12
Mahmud Z, Tikunova S, Belevych N, Wagg CS, Zhabyeyev P, Liu PB, Rasicci DV, Yengo CM, Oudit GY, Lopaschuk GD, Reiser PJ, Davis JP, Hwang PM. Small Molecule RPI-194 Stabilizes Activated Troponin to Increase the Calcium Sensitivity of Striated Muscle Contraction. Front Physiol 2022;13:892979. [PMID: 35755445 PMCID: PMC9213791 DOI: 10.3389/fphys.2022.892979] [Citation(s) in RCA: 5] [Impact Index Per Article: 1.7] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 03/09/2022] [Accepted: 05/10/2022] [Indexed: 11/13/2022]  Open
13
杨 东. Recent research on the treatment of spinal muscular atrophy. ZHONGGUO DANG DAI ER KE ZA ZHI = CHINESE JOURNAL OF CONTEMPORARY PEDIATRICS 2022;24:204-209. [PMID: 35209987 PMCID: PMC8884051 DOI: 10.7499/j.issn.1008-8830.2110041] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Subscribe] [Academic Contribution Register] [Received: 10/12/2021] [Accepted: 12/20/2021] [Indexed: 01/24/2023]
14
Aslesh T, Yokota T. Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy. Cells 2022;11:417. [PMID: 35159227 PMCID: PMC8834523 DOI: 10.3390/cells11030417] [Citation(s) in RCA: 32] [Impact Index Per Article: 10.7] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 12/20/2021] [Revised: 01/14/2022] [Accepted: 01/24/2022] [Indexed: 02/06/2023]  Open
15
Edinoff AN, Nguyen LH, Odisho AS, Maxey BS, Pruitt JW, Girma B, Cornett EM, Kaye AM, Kaye AD. The Antisense Oligonucleotide Nusinersen for Treatment of Spinal Muscular Atrophy. Orthop Rev (Pavia) 2021;13:24934. [PMID: 34745470 DOI: 10.52965/001c.24934] [Citation(s) in RCA: 14] [Impact Index Per Article: 3.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Received: 06/02/2021] [Accepted: 06/17/2021] [Indexed: 01/25/2023]  Open
16
Chong LC, Gandhi G, Lee JM, Yeo WWY, Choi SB. Drug Discovery of Spinal Muscular Atrophy (SMA) from the Computational Perspective: A Comprehensive Review. Int J Mol Sci 2021;22:8962. [PMID: 34445667 PMCID: PMC8396480 DOI: 10.3390/ijms22168962] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/15/2021] [Accepted: 01/27/2021] [Indexed: 01/02/2023]  Open
17
Rekand IH, Brenk R. DrugPred_RNA-A Tool for Structure-Based Druggability Predictions for RNA Binding Sites. J Chem Inf Model 2021;61:4068-4081. [PMID: 34286972 PMCID: PMC8389535 DOI: 10.1021/acs.jcim.1c00155] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 01/14/2023]
18
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
19
Axford J, Sung MJ, Manchester J, Chin D, Jain M, Shin Y, Dix I, Hamann LG, Cheung AK, Sivasankaran R, Briner K, Dales NA, Hurley B. Use of Intramolecular 1,5-Sulfur-Oxygen and 1,5-Sulfur-Halogen Interactions in the Design of N-Methyl-5-aryl-N-(2,2,6,6-tetramethylpiperidin-4-yl)-1,3,4-thiadiazol-2-amine SMN2 Splicing Modulators. J Med Chem 2021;64:4744-4761. [PMID: 33822618 DOI: 10.1021/acs.jmedchem.0c02173] [Citation(s) in RCA: 9] [Impact Index Per Article: 2.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/17/2022]
20
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
21
Meyer SM, Williams CC, Akahori Y, Tanaka T, Aikawa H, Tong Y, Childs-Disney JL, Disney MD. Small molecule recognition of disease-relevant RNA structures. Chem Soc Rev 2020;49:7167-7199. [PMID: 32975549 PMCID: PMC7717589 DOI: 10.1039/d0cs00560f] [Citation(s) in RCA: 102] [Impact Index Per Article: 20.4] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/14/2022]
22
Masoudi M, Bereshneh AH, Rasoulinezhad M, Ashrafi MR, Garshasbi M, Tavasoli AR. Leukoencephalopathy in Al-Raqad syndrome: Expanding the clinical and neuroimaging features caused by a biallelic novel missense variant in DCPS. Am J Med Genet A 2020;182:2391-2398. [PMID: 32770650 DOI: 10.1002/ajmg.a.61776] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 12/26/2019] [Revised: 05/05/2020] [Accepted: 05/07/2020] [Indexed: 11/11/2022]
23
Wadman RI, Jansen MD, Stam M, Wijngaarde CA, Curial CAD, Medic J, Sodaar P, Schouten J, Vijzelaar R, Lemmink HH, van den Berg LH, Groen EJN, van der Pol WL. Intragenic and structural variation in the SMN locus and clinical variability in spinal muscular atrophy. Brain Commun 2020;2:fcaa075. [PMID: 32954327 PMCID: PMC7425299 DOI: 10.1093/braincomms/fcaa075] [Citation(s) in RCA: 38] [Impact Index Per Article: 7.6] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 02/19/2020] [Revised: 04/17/2020] [Accepted: 04/22/2020] [Indexed: 11/15/2022]  Open
24
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
25
Ross LF, Kwon JM. Spinal Muscular Atrophy: Past, Present, and Future. Neoreviews 2020;20:e437-e451. [PMID: 31371553 DOI: 10.1542/neo.20-8-e437] [Citation(s) in RCA: 26] [Impact Index Per Article: 5.2] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 01/05/2023]
26
RNA-Targeted Therapies and High-Throughput Screening Methods. Int J Mol Sci 2020;21:ijms21082996. [PMID: 32340368 PMCID: PMC7216119 DOI: 10.3390/ijms21082996] [Citation(s) in RCA: 17] [Impact Index Per Article: 3.4] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 03/31/2020] [Revised: 04/20/2020] [Accepted: 04/21/2020] [Indexed: 02/07/2023]  Open
27
Swalley SE. Expanding therapeutic opportunities for neurodegenerative diseases: A perspective on the important role of phenotypic screening. Bioorg Med Chem 2020;28:115239. [DOI: 10.1016/j.bmc.2019.115239] [Citation(s) in RCA: 9] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 08/02/2019] [Revised: 11/15/2019] [Accepted: 11/24/2019] [Indexed: 02/08/2023]
28
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]
29
Unveiling the druggable RNA targets and small molecule therapeutics. Bioorg Med Chem 2019;27:2149-2165. [PMID: 30981606 PMCID: PMC7126819 DOI: 10.1016/j.bmc.2019.03.057] [Citation(s) in RCA: 29] [Impact Index Per Article: 4.8] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 12/17/2018] [Revised: 03/25/2019] [Accepted: 03/29/2019] [Indexed: 12/15/2022]
30
Long KK, O’Shea KM, Khairallah RJ, Howell K, Paushkin S, Chen KS, Cote SM, Webster MT, Stains JP, Treece E, Buckler A, Donovan A. Specific inhibition of myostatin activation is beneficial in mouse models of SMA therapy. Hum Mol Genet 2019;28:1076-1089. [PMID: 30481286 PMCID: PMC6423420 DOI: 10.1093/hmg/ddy382] [Citation(s) in RCA: 79] [Impact Index Per Article: 13.2] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 06/29/2018] [Revised: 10/29/2018] [Accepted: 10/31/2018] [Indexed: 12/22/2022]  Open
31
Intraperitoneal delivery of a novel drug-like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy. Sci Rep 2019;9:1633. [PMID: 30733501 PMCID: PMC6367425 DOI: 10.1038/s41598-018-38208-9] [Citation(s) in RCA: 8] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 10/25/2018] [Accepted: 12/20/2018] [Indexed: 01/08/2023]  Open
32
Ratni H, Mueller L, Ebeling M. Rewriting the (tran)script: Application to spinal muscular atrophy. PROGRESS IN MEDICINAL CHEMISTRY 2019;58:119-156. [PMID: 30879473 DOI: 10.1016/bs.pmch.2018.12.003] [Citation(s) in RCA: 5] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Academic Contribution Register] [Indexed: 06/09/2023]
33
Warner KD, Hajdin CE, Weeks KM. Principles for targeting RNA with drug-like small molecules. Nat Rev Drug Discov 2018;17:547-558. [PMID: 29977051 PMCID: PMC6420209 DOI: 10.1038/nrd.2018.93] [Citation(s) in RCA: 458] [Impact Index Per Article: 65.4] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/11/2022]
34
Markossian S, Ang KK, Wilson CG, Arkin MR. Small-Molecule Screening for Genetic Diseases. Annu Rev Genomics Hum Genet 2018;19:263-288. [PMID: 29799800 DOI: 10.1146/annurev-genom-083117-021452] [Citation(s) in RCA: 9] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 11/09/2022]
35
Osman EY, Washington CW, Simon ME, Megiddo D, Greif H, Lorson CL. Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular Atrophy. J Neuromuscul Dis 2018;4:237-249. [PMID: 28598854 DOI: 10.3233/jnd-170230] [Citation(s) in RCA: 14] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Abstract] [Key Words] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/13/2022]
36
Edwards JD, Butchbach MER. Effect of the Butyrate Prodrug Pivaloyloxymethyl Butyrate (AN9) on a Mouse Model for Spinal Muscular Atrophy. J Neuromuscul Dis 2018;3:511-515. [PMID: 27911337 DOI: 10.3233/jnd-160187] [Citation(s) in RCA: 6] [Impact Index Per Article: 0.9] [Reference Citation Analysis] [Abstract] [Key Words] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 01/07/2023]
37
Alternative Splicing in the Hippo Pathway-Implications for Disease and Potential Therapeutic Targets. Genes (Basel) 2018. [PMID: 29534050 PMCID: PMC5867882 DOI: 10.3390/genes9030161] [Citation(s) in RCA: 11] [Impact Index Per Article: 1.6] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/14/2022]  Open
38
Parente V, Corti S. Advances in spinal muscular atrophy therapeutics. Ther Adv Neurol Disord 2018;11:1756285618754501. [PMID: 29434670 PMCID: PMC5802612 DOI: 10.1177/1756285618754501] [Citation(s) in RCA: 53] [Impact Index Per Article: 7.6] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 05/21/2017] [Accepted: 10/24/2017] [Indexed: 11/17/2022]  Open
39
Small-molecule flunarizine increases SMN protein in nuclear Cajal bodies and motor function in a mouse model of spinal muscular atrophy. Sci Rep 2018;8:2075. [PMID: 29391529 PMCID: PMC5794986 DOI: 10.1038/s41598-018-20219-1] [Citation(s) in RCA: 6] [Impact Index Per Article: 0.9] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 09/06/2017] [Accepted: 01/16/2018] [Indexed: 02/07/2023]  Open
40
Khadilkar SV, Yadav RS, Patel BA. Spinal Muscular Atrophy. Neuromuscul Disord 2018. [DOI: 10.1007/978-981-10-5361-0_10] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 10/18/2022]
41
Choi S, Calder AN, Miller EH, Anderson KP, Fiejtek DK, Rietz A, Li H, Cherry JJ, Quist KM, Xing X, Glicksman MA, Cuny GD, Lorson CL, Androphy EA, Hodgetts KJ. Optimization of a series of heterocycles as survival motor neuron gene transcription enhancers. Bioorg Med Chem Lett 2017;27:5144-5148. [PMID: 29103974 PMCID: PMC5701662 DOI: 10.1016/j.bmcl.2017.10.066] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 09/02/2017] [Revised: 10/22/2017] [Accepted: 10/24/2017] [Indexed: 12/24/2022]
42
RNA as a small molecule druggable target. Bioorg Med Chem Lett 2017;27:5083-5088. [PMID: 29097169 DOI: 10.1016/j.bmcl.2017.10.052] [Citation(s) in RCA: 53] [Impact Index Per Article: 6.6] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 08/11/2017] [Revised: 10/11/2017] [Accepted: 10/22/2017] [Indexed: 12/20/2022]
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Bharucha-Goebel D, Kaufmann P. Treatment Advances in Spinal Muscular Atrophy. Curr Neurol Neurosci Rep 2017;17:91. [PMID: 28983837 DOI: 10.1007/s11910-017-0798-y] [Citation(s) in RCA: 27] [Impact Index Per Article: 3.4] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 01/03/2023]
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Vauthier V, Housset C, Falguières T. Targeted pharmacotherapies for defective ABC transporters. Biochem Pharmacol 2017;136:1-11. [DOI: 10.1016/j.bcp.2017.02.020] [Citation(s) in RCA: 29] [Impact Index Per Article: 3.6] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/24/2017] [Accepted: 02/23/2017] [Indexed: 02/07/2023]
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Therapeutic approaches for spinal muscular atrophy (SMA). Gene Ther 2017;24:514-519. [DOI: 10.1038/gt.2017.45] [Citation(s) in RCA: 39] [Impact Index Per Article: 4.9] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 03/08/2017] [Accepted: 05/09/2017] [Indexed: 12/13/2022]
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Salameh A, Keller M, Dähnert I, Dhein S. Olesoxime Inhibits Cardioplegia-Induced Ischemia/Reperfusion Injury. A Study in Langendorff-Perfused Rabbit Hearts. Front Physiol 2017;8:324. [PMID: 28579963 PMCID: PMC5437207 DOI: 10.3389/fphys.2017.00324] [Citation(s) in RCA: 9] [Impact Index Per Article: 1.1] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/06/2017] [Accepted: 05/05/2017] [Indexed: 12/15/2022]  Open
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Rietz A, Li H, Quist KM, Cherry JJ, Lorson CL, Burnett BG, Kern NL, Calder AN, Fritsche M, Lusic H, Boaler PJ, Choi S, Xing X, Glicksman MA, Cuny GD, Androphy EJ, Hodgetts KJ. Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy. J Med Chem 2017;60:4594-4610. [PMID: 28481536 DOI: 10.1021/acs.jmedchem.6b01885] [Citation(s) in RCA: 11] [Impact Index Per Article: 1.4] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 01/29/2023]
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Pinard E, Green L, Reutlinger M, Weetall M, Naryshkin NA, Baird J, Chen KS, Paushkin SV, Metzger F, Ratni H. Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy. J Med Chem 2017;60:4444-4457. [DOI: 10.1021/acs.jmedchem.7b00406] [Citation(s) in RCA: 20] [Impact Index Per Article: 2.5] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 01/24/2023]
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Farrelly-Rosch A, Lau CL, Patil N, Turner BJ, Shabanpoor F. Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts. Neurochem Int 2017;108:213-221. [PMID: 28389270 DOI: 10.1016/j.neuint.2017.02.016] [Citation(s) in RCA: 12] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Key Words] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/23/2017] [Revised: 02/22/2017] [Accepted: 02/24/2017] [Indexed: 12/16/2022]
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Gopalsamy A, Narayanan A, Liu S, Parikh MD, Kyne RE, Fadeyi O, Tones MA, Cherry JJ, Nabhan JF, LaRosa G, Petersen DN, Menard C, Foley TL, Noell S, Ren Y, Loria PM, Maglich-Goodwin J, Rong H, Jones LH. Design of Potent mRNA Decapping Scavenger Enzyme (DcpS) Inhibitors with Improved Physicochemical Properties To Investigate the Mechanism of Therapeutic Benefit in Spinal Muscular Atrophy (SMA). J Med Chem 2017;60:3094-3108. [DOI: 10.1021/acs.jmedchem.7b00124] [Citation(s) in RCA: 16] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 01/27/2023]
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