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For: Prasad V. Nusinersen for Spinal Muscular Atrophy: Are We Paying Too Much for Too Little? JAMA Pediatr 2018;172:123-125. [PMID: 29228077 DOI: 10.1001/jamapediatrics.2017.4360] [Citation(s) in RCA: 21] [Impact Index Per Article: 3.0] [Reference Citation Analysis] [What about the content of this article? (0)] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 12/22/2022]
Number Cited by Other Article(s)
1
Romano R, Bucci C. Antisense therapy: a potential breakthrough in the treatment of neurodegenerative diseases. Neural Regen Res 2024;19:1027-1035. [PMID: 37862205 PMCID: PMC10749614 DOI: 10.4103/1673-5374.385285] [Citation(s) in RCA: 2] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 04/17/2023] [Revised: 06/13/2023] [Accepted: 07/21/2023] [Indexed: 10/22/2023]  Open
2
Jiao Y, Zhao J, Wang Z, Chen X, Cai H, Huang X, Sun P, Shen J, Song F, Xiong H, Dai Y, Chen W, Shen J. How do orphan disease patients live during the pandemic of Omicron variant? A nationwide survey of spinal muscular atrophy patients in China. Int J Infect Dis 2023;134:187-194. [PMID: 37352912 DOI: 10.1016/j.ijid.2023.06.015] [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: 02/10/2023] [Revised: 05/16/2023] [Accepted: 06/17/2023] [Indexed: 06/25/2023]  Open
3
Ingle RG, Fang WJ. An Overview of the Stability and Delivery Challenges of Commercial Nucleic Acid Therapeutics. Pharmaceutics 2023;15:pharmaceutics15041158. [PMID: 37111643 PMCID: PMC10143938 DOI: 10.3390/pharmaceutics15041158] [Citation(s) in RCA: 8] [Impact Index Per Article: 4.0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/20/2023] [Revised: 03/21/2023] [Accepted: 03/24/2023] [Indexed: 04/29/2023]  Open
4
Anwar S, Mir F, Yokota T. Enhancing the Effectiveness of Oligonucleotide Therapeutics Using Cell-Penetrating Peptide Conjugation, Chemical Modification, and Carrier-Based Delivery Strategies. Pharmaceutics 2023;15:pharmaceutics15041130. [PMID: 37111616 PMCID: PMC10140998 DOI: 10.3390/pharmaceutics15041130] [Citation(s) in RCA: 15] [Impact Index Per Article: 7.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 01/20/2023] [Revised: 03/28/2023] [Accepted: 03/28/2023] [Indexed: 04/29/2023]  Open
5
Nanomedicine based strategies for oligonucleotide traversion across the blood-brain barrier. J Control Release 2023;354:554-571. [PMID: 36649742 DOI: 10.1016/j.jconrel.2023.01.031] [Citation(s) in RCA: 5] [Impact Index Per Article: 2.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 12/06/2022] [Revised: 01/10/2023] [Accepted: 01/12/2023] [Indexed: 01/19/2023]
6
Djordjevic D, McFadyen A, Anderson JA. Ethical challenges and opportunities in the development and approval of novel therapeutics for rare diseases. THE JOURNAL OF MEDICINE ACCESS 2023;7:27550834231177507. [PMID: 37323852 PMCID: PMC10262601 DOI: 10.1177/27550834231177507] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Subscribe] [Academic Contribution Register] [Received: 01/25/2023] [Accepted: 05/05/2023] [Indexed: 06/17/2023]
7
Carey KA, Farrar MA, Kasparian NA, Street DJ, De Abreu Lourenco R. Family, healthcare professional, and societal preferences for the treatment of infantile spinal muscular atrophy: A discrete choice experiment. Dev Med Child Neurol 2022;64:753-761. [PMID: 34962299 DOI: 10.1111/dmcn.15135] [Citation(s) in RCA: 5] [Impact Index Per Article: 1.7] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Received: 07/13/2021] [Revised: 11/09/2021] [Accepted: 11/16/2021] [Indexed: 11/29/2022]
8
Blonda A, Barcina Lacosta T, Toumi M, Simoens S. Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries. Front Pharmacol 2022;12:750742. [PMID: 35126102 PMCID: PMC8814578 DOI: 10.3389/fphar.2021.750742] [Citation(s) in RCA: 7] [Impact Index Per Article: 2.3] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 07/31/2021] [Accepted: 09/30/2021] [Indexed: 11/13/2022]  Open
9
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
10
Hiebeler M, Abicht A, Reilich P, Walter MC. Effect of Discontinuation of Nusinersen Treatment in Long-Standing SMA3. J Neuromuscul Dis 2021;8:537-542. [PMID: 33682724 DOI: 10.3233/jnd-210644] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 11/15/2022]
11
Jalali A, Rothwell E, Botkin JR, Anderson RA, Butterfield RJ, Nelson RE. Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy. J Pediatr 2020;227:274-280.e2. [PMID: 32659229 PMCID: PMC7686158 DOI: 10.1016/j.jpeds.2020.07.033] [Citation(s) in RCA: 17] [Impact Index Per Article: 3.4] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Received: 01/08/2020] [Revised: 07/03/2020] [Accepted: 07/08/2020] [Indexed: 12/22/2022]
12
Le TK, Paris C, Khan KS, Robson F, Ng WL, Rocchi P. Nucleic Acid-Based Technologies Targeting Coronaviruses. Trends Biochem Sci 2020;46:351-365. [PMID: 33309323 PMCID: PMC7691141 DOI: 10.1016/j.tibs.2020.11.010] [Citation(s) in RCA: 19] [Impact Index Per Article: 3.8] [Reference Citation Analysis] [Abstract] [Key Words] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 09/11/2020] [Revised: 11/19/2020] [Accepted: 11/24/2020] [Indexed: 12/12/2022]
13
Chua KP, Conti RM. Trends In Orphan Drug Spending And Out-Of-Pocket Spending Among US Children, 2013-18. Health Aff (Millwood) 2020;39:1806-1811. [PMID: 33017253 DOI: 10.1377/hlthaff.2020.00595] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 11/05/2022]
14
Advances in oligonucleotide drug delivery. Nat Rev Drug Discov 2020;19:673-694. [PMID: 32782413 PMCID: PMC7419031 DOI: 10.1038/s41573-020-0075-7] [Citation(s) in RCA: 1150] [Impact Index Per Article: 230.0] [Reference Citation Analysis] [Abstract] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Accepted: 06/24/2020] [Indexed: 12/12/2022]
15
Song JY, Kim HS, Park SJ, Lee J, Lee J. Nusinersen Administration in Spinal Muscular Atrophy Patients with Severe Scoliosis: Interlaminar Approaches at the Lumbar Level. ANNALS OF CHILD NEUROLOGY 2020. [DOI: 10.26815/acn.2020.00045] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.4] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Academic Contribution Register] [Indexed: 12/22/2022]  Open
16
“The Whole Game is Changing and You’ve Got Hope”: Australian Perspectives on Treatment Decision Making in Spinal Muscular Atrophy. PATIENT-PATIENT CENTERED OUTCOMES RESEARCH 2020;13:389-400. [DOI: 10.1007/s40271-020-00415-w] [Citation(s) in RCA: 9] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Track Full Text] [Subscribe] [Academic Contribution Register] [Indexed: 12/28/2022]
17
Droege M, Sproule D, Arjunji R, Gauthier-Loiselle M, Cloutier M, Dabbous O. Economic burden of spinal muscular atrophy in the United States: a contemporary assessment. J Med Econ 2020;23:70-79. [PMID: 31322019 DOI: 10.1080/13696998.2019.1646263] [Citation(s) in RCA: 20] [Impact Index Per Article: 4.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 12/22/2022]
18
Hoot NR. Nusinersen for Type 1 Spinal Muscular Atrophy: A Father's Perspective. Pediatrics 2019;144:peds.2019-0226. [PMID: 31506303 DOI: 10.1542/peds.2019-0226] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Accepted: 04/22/2019] [Indexed: 11/24/2022]  Open
19
Sequencing as a first-line methodology for cystic fibrosis carrier screening. Genet Med 2019;21:2569-2576. [PMID: 31036917 PMCID: PMC6831513 DOI: 10.1038/s41436-019-0525-y] [Citation(s) in RCA: 18] [Impact Index Per Article: 3.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 02/13/2019] [Accepted: 04/16/2019] [Indexed: 12/18/2022]  Open
20
Gidaro T, Servais L. Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps. Dev Med Child Neurol 2019;61:19-24. [PMID: 30221755 DOI: 10.1111/dmcn.14027] [Citation(s) in RCA: 91] [Impact Index Per Article: 15.2] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Accepted: 07/25/2018] [Indexed: 01/08/2023]
21
Ravenscroft G, Bryson-Richardson RJ, Nowak KJ, Laing NG. Recent advances in understanding congenital myopathies. F1000Res 2018;7. [PMID: 30631434 PMCID: PMC6290972 DOI: 10.12688/f1000research.16422.1] [Citation(s) in RCA: 18] [Impact Index Per Article: 2.6] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Accepted: 11/29/2018] [Indexed: 12/18/2022]  Open
22
Sansone VA, Albamonte E, Salmin F, Casiraghi J, Pirola A, Bettinelli M, Rao F, Mancini L, Tovaglieri N, Fedeli F, Stoia P, Heinen M, Cozzi V, Carraro E, Lunetta C, Di Bari A, Mercuri E. Intrathecal nusinersen treatment for SMA in a dedicated neuromuscular clinic: an example of multidisciplinary and integrated care. Neurol Sci 2018;40:327-332. [PMID: 30430317 DOI: 10.1007/s10072-018-3622-9] [Citation(s) in RCA: 14] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Abstract] [Key Words] [Journal Information] [Subscribe] [Academic Contribution Register] [Received: 08/09/2018] [Accepted: 10/24/2018] [Indexed: 11/29/2022]
23
Missing Potential Conflict of Interest. JAMA Pediatr 2018;172:891. [PMID: 30073265 DOI: 10.1001/jamapediatrics.2018.2493] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 11/14/2022]
24
Prasad V. Inconsistent Reporting of Potential Conflicts of Interest. JAMA Pediatr 2018;172:886. [PMID: 30073291 DOI: 10.1001/jamapediatrics.2018.2420] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 11/14/2022]
25
Prasad V. Cost-effectiveness of Nusinersen for Spinal Muscular Atrophy-Reply. JAMA Pediatr 2018;172:701-702. [PMID: 29800980 DOI: 10.1001/jamapediatrics.2018.0733] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.1] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 11/14/2022]
26
Richardson RC. Cost-effectiveness of Nusinersen for Spinal Muscular Atrophy. JAMA Pediatr 2018;172:701. [PMID: 29801053 DOI: 10.1001/jamapediatrics.2018.0772] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Academic Contribution Register] [Indexed: 11/14/2022]
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