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 Custom Filters

Results by year

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

Year Number of Results
1911 2
1913 1
1917 2
1945 2
1946 7
1947 4
1948 5
1949 4
1950 8
1951 14
1952 19
1953 21
1954 21
1955 14
1956 16
1957 15
1958 29
1959 8
1960 4
1961 7
1962 13
1963 15
1964 7
1965 3
1966 9
1967 7
1968 8
1969 9
1970 11
1971 17
1972 5
1973 15
1974 9
1975 15
1976 20
1977 17
1978 20
1979 16
1980 20
1981 24
1982 19
1983 23
1984 28
1985 24
1986 26
1987 43
1988 81
1989 100
1990 77
1991 61
1992 77
1993 117
1994 96
1995 106
1996 142
1997 128
1998 98
1999 120
2000 119
2001 140
2002 151
2003 150
2004 139
2005 143
2006 132
2007 179
2008 193
2009 201
2010 244
2011 267
2012 298
2013 291
2014 322
2015 316
2016 304
2017 388
2018 426
2019 461
2020 624
2021 637
2022 609
2023 648
2024 690
2025 60

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

9,053 results

Results by year

Citations

1 article found by citation matching

Search results

Filters applied: . Clear all
Page 1
Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma).
Ogbonmide T, Rathore R, Rangrej SB, Hutchinson S, Lewis M, Ojilere S, Carvalho V, Kelly I. Ogbonmide T, et al. Cureus. 2023 Mar 15;15(3):e36197. doi: 10.7759/cureus.36197. eCollection 2023 Mar. Cureus. 2023. PMID: 37065340 Free PMC article. Review.
Spinal Muscular Atrophy (SMA) is a genetic disease that causes weakness and wasting in the voluntary muscles of infants and children. ...For this, we have conducted a literature search on PubMed, MEDLINE, and Ovid (2019 to 2022) in the English language using …
Spinal Muscular Atrophy (SMA) is a genetic disease that causes weakness and wasting in the voluntary muscles of infants …
Spinal Muscular Atrophy: The Past, Present, and Future of Diagnosis and Treatment.
Nishio H, Niba ETE, Saito T, Okamoto K, Takeshima Y, Awano H. Nishio H, et al. Int J Mol Sci. 2023 Jul 26;24(15):11939. doi: 10.3390/ijms241511939. Int J Mol Sci. 2023. PMID: 37569314 Free PMC article. Review.
Spinal muscular atrophy (SMA) is a lower motor neuron disease with autosomal recessive inheritance. ...Two other drugs were subsequently approved: onasemnogene abeparvovec and risdiplam. Clinical trials with these drugs targeting patients with pre-symp …
Spinal muscular atrophy (SMA) is a lower motor neuron disease with autosomal recessive inheritance. ...Two other drugs …
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, Iannaccone ST, Kuntz NL, Peña LDM, Shieh PB, Smith EC, Kwon JM, Zaidman CM, Schultz M, Feltner DE, Tauscher-Wisniewski S, Ouyang H, Chand DH, Sproule DM, Macek TA, Mendell JR. Day JW, et al. Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17. Lancet Neurol. 2021. PMID: 33743238 Clinical Trial.
BACKGROUND: Spinal muscular atrophy type 1 is a motor neuron disorder resulting in death or the need for permanent ventilation by age 2 years. ...The favourable benefit-risk profile shown in this study supports the use of onasemnogene abeparvovec for t …
BACKGROUND: Spinal muscular atrophy type 1 is a motor neuron disorder resulting in death or the need for permanent vent …
Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy.
Blair HA. Blair HA. CNS Drugs. 2022 Sep;36(9):995-1005. doi: 10.1007/s40263-022-00941-1. Epub 2022 Aug 12. CNS Drugs. 2022. PMID: 35960489 Review.
Onasemnogene abeparvovec (Zolgensma()) is a gene therapy approved for the treatment of spinal muscular atrophy (SMA). ...Spinal muscular atrophy (SMA) is a rare genetic condition that causes muscle weakness and wasting. ...
Onasemnogene abeparvovec (Zolgensma()) is a gene therapy approved for the treatment of spinal muscular atroph …
Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data.
Erdos J, Wild C. Erdos J, et al. Eur J Paediatr Neurol. 2022 Jul;39:1-10. doi: 10.1016/j.ejpn.2022.04.006. Epub 2022 Apr 30. Eur J Paediatr Neurol. 2022. PMID: 35533607 Free article. Review.
OBJECTIVES: This systematic review aimed to assess mid- and long-term (at least 12 months) real-world study data from all types of spinal muscular atrophy (SMA) patients treated with any of the approved drugs or combination therapies. ...RESULTS: Five hundred …
OBJECTIVES: This systematic review aimed to assess mid- and long-term (at least 12 months) real-world study data from all types of spinal …
Spinal Muscular Atrophy.
Nicolau S, Waldrop MA, Connolly AM, Mendell JR. Nicolau S, et al. Semin Pediatr Neurol. 2021 Apr;37:100878. doi: 10.1016/j.spen.2021.100878. Epub 2021 Feb 11. Semin Pediatr Neurol. 2021. PMID: 33892848 Review.
Spinal muscular atrophy is one of the most common neuromuscular disorders of childhood and has high morbidity and mortality. ...Newborn screening programs are enabling earlier diagnosis and treatment and better outcomes, but respiratory care and other support …
Spinal muscular atrophy is one of the most common neuromuscular disorders of childhood and has high morbidity and morta …
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis.
Pascual-Morena C, Cavero-Redondo I, Lucerón-Lucas-Torres M, Martínez-García I, Rodríguez-Gutiérrez E, Martínez-Vizcaíno V. Pascual-Morena C, et al. Hum Gene Ther. 2023 Feb;34(3-4):129-138. doi: 10.1089/hum.2022.161. Hum Gene Ther. 2023. PMID: 36136906 Review.
One of the latest approved therapies for spinal muscular atrophy (SMA) is onasemnogene abeparvovec, which transduces motor neurons with the survival of motor neuron gene. The aim of this meta-analysis was to estimate the effect of onasemnogene a …
One of the latest approved therapies for spinal muscular atrophy (SMA) is onasemnogene abeparvovec, which transd …
Spinal Muscular Atrophy.
Oskoui M, Servais L. Oskoui M, et al. Continuum (Minneap Minn). 2023 Oct 1;29(5):1564-1584. doi: 10.1212/CON.0000000000001338. Continuum (Minneap Minn). 2023. PMID: 37851043 Review.
OBJECTIVE: This article provides a comprehensive overview of the diagnostic assessment and treatment of individuals with spinal muscular atrophy (SMA) due to homozygous deletions of SMN1 . LATEST DEVELOPMENTS: In recent years, most states have incorporated SM …
OBJECTIVE: This article provides a comprehensive overview of the diagnostic assessment and treatment of individuals with spinal mu …
Spinal Muscular Atrophy.
Prior TW, Leach ME, Finanger EL. Prior TW, et al. 2000 Feb 24 [updated 2024 Sep 19]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2025. 2000 Feb 24 [updated 2024 Sep 19]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2025. PMID: 20301526 Free Books & Documents. Review.
CLINICAL CHARACTERISTICS: Spinal muscular atrophy (SMA) is characterized by muscle weakness and atrophy resulting from progressive degeneration and irreversible loss of the anterior horn cells in the spinal cord (i.e., lower motor neurons) and the brain stem …
CLINICAL CHARACTERISTICS: Spinal muscular atrophy (SMA) is characterized by muscle weakness and atrophy resulting from …
Advances and limitations for the treatment of spinal muscular atrophy.
Day JW, Howell K, Place A, Long K, Rossello J, Kertesz N, Nomikos G. Day JW, et al. BMC Pediatr. 2022 Nov 3;22(1):632. doi: 10.1186/s12887-022-03671-x. BMC Pediatr. 2022. PMID: 36329412 Free PMC article. Review.
Spinal muscular atrophy (5q-SMA; SMA), a genetic neuromuscular condition affecting spinal motor neurons, is caused by defects in both copies of the SMN1 gene that produces survival motor neuron (SMN) protein. ...While the therapeutic approaches with Spinraza( …
Spinal muscular atrophy (5q-SMA; SMA), a genetic neuromuscular condition affecting spinal motor neurons, is caused by d …
9,053 results