Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Results by year

Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1993 1
2001 1
2002 3
2003 3
2004 2
2005 2
2006 3
2007 3
2008 3
2009 7
2010 5
2011 10
2012 9
2013 11
2014 14
2015 11
2016 8
2017 10
2018 8
2019 10
2020 9
2021 9
2022 6
2023 3
2024 1

Text availability

Article attribute

Article type

Publication date

Search Results

135 results

Results by year

Filters applied: . Clear all
Page 1
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy.
Alves CRR, Ha LL, Yaworski R, Sutton ER, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP. Alves CRR, et al. Among authors: swoboda kj. Nat Biomed Eng. 2024 Feb;8(2):118-131. doi: 10.1038/s41551-023-01132-z. Epub 2023 Dec 6. Nat Biomed Eng. 2024. PMID: 38057426 Free PMC article.
Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study.
Crawford TO, Swoboda KJ, De Vivo DC, Bertini E, Hwu WL, Finkel RS, Kirschner J, Kuntz NL, Nazario AN, Parsons JA, Pechmann A, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Zhu C, Raynaud S, Lago TR, Paradis AD, Foster R, Chin R, Berger Z; NURTURE Study Group. Crawford TO, et al. Among authors: swoboda kj. Muscle Nerve. 2023 Aug;68(2):157-170. doi: 10.1002/mus.27853. Epub 2023 Jul 6. Muscle Nerve. 2023. PMID: 37409780
Base editing as a genetic treatment for spinal muscular atrophy.
Alves CRR, Ha LL, Yaworski R, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP. Alves CRR, et al. Among authors: swoboda kj. bioRxiv [Preprint]. 2023 Jan 21:2023.01.20.524978. doi: 10.1101/2023.01.20.524978. bioRxiv. 2023. PMID: 36711797 Free PMC article. Preprint.
De novo variants in genes regulating stress granule assembly associate with neurodevelopmental disorders.
Jia X, Zhang S, Tan S, Du B, He M, Qin H, Chen J, Duan X, Luo J, Chen F, Ouyang L, Wang J, Chen G, Yu B, Zhang G, Zhang Z, Lyu Y, Huang Y, Jiao J, Chen JYH, Swoboda KJ, Agolini E, Novelli A, Leoni C, Zampino G, Cappuccio G, Brunetti-Pierri N, Gerard B, Ginglinger E, Richer J, McMillan H, White-Brown A, Hoekzema K, Bernier RA, Kurtz-Nelson EC, Earl RK, Meddens C, Alders M, Fuchs M, Caumes R, Brunelle P, Smol T, Kuehl R, Day-Salvatore DL, Monaghan KG, Morrow MM, Eichler EE, Hu Z, Yuan L, Tan J, Xia K, Shen Y, Guo H. Jia X, et al. Among authors: swoboda kj. Sci Adv. 2022 Aug 19;8(33):eabo7112. doi: 10.1126/sciadv.abo7112. Epub 2022 Aug 17. Sci Adv. 2022. PMID: 35977029 Free PMC article.
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.
Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, McMillan HJ, Finkel RS, Swoboda KJ, Kwon JM, Zaidman CM, Chiriboga CA, Iannaccone ST, Krueger JM, Parsons JA, Shieh PB, Kavanagh S, Wigderson M, Tauscher-Wisniewski S, McGill BE, Macek TA. Strauss KA, et al. Among authors: swoboda kj. Nat Med. 2022 Jul;28(7):1390-1397. doi: 10.1038/s41591-022-01867-3. Epub 2022 Jun 17. Nat Med. 2022. PMID: 35715567 Free PMC article. Clinical Trial.
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, McMillan HJ, Finkel RS, Swoboda KJ, Kwon JM, Zaidman CM, Chiriboga CA, Iannaccone ST, Krueger JM, Parsons JA, Shieh PB, Kavanagh S, Tauscher-Wisniewski S, McGill BE, Macek TA. Strauss KA, et al. Among authors: swoboda kj. Nat Med. 2022 Jul;28(7):1381-1389. doi: 10.1038/s41591-022-01866-4. Epub 2022 Jun 17. Nat Med. 2022. PMID: 35715566 Free PMC article. Clinical Trial.
An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.
Kwon JM, Arya K, Kuntz N, Phan HC, Sieburg C, Swoboda KJ, Veerapandiyan A, Assman B, Bader-Weder S, Dickendesher TL, Hansen J, Lin H, Yan Y, Rao VK; US Expanded Access Program Working Group. Kwon JM, et al. Among authors: swoboda kj. Ann Clin Transl Neurol. 2022 Jun;9(6):810-818. doi: 10.1002/acn3.51560. Epub 2022 May 14. Ann Clin Transl Neurol. 2022. PMID: 35567422 Free PMC article.
Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients.
Magen I, Aharoni S, Yacovzada NS, Tokatly Latzer I, Alves CRR, Sagi L, Fattal-Valevski A, Swoboda KJ, Katz J, Bruckheimer E, Nevo Y, Hornstein E. Magen I, et al. Among authors: swoboda kj. Eur J Neurol. 2022 Aug;29(8):2420-2430. doi: 10.1111/ene.15382. Epub 2022 Jun 7. Eur J Neurol. 2022. PMID: 35510740 Free PMC article.
Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series.
Alves CRR, Petrillo M, Spellman R, Garner R, Zhang R, Kiefer M, Simeone S, Sohn J, Eichelberger EJ, Rodrigues E, Arruda EA, Townsend EL, Farwell W, Swoboda KJ. Alves CRR, et al. Among authors: swoboda kj. Mol Ther Methods Clin Dev. 2021 Oct 30;23:524-538. doi: 10.1016/j.omtm.2021.10.011. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34853799 Free PMC article.
135 results