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
2011 5
2012 3
2013 6
2014 6
2015 5
2016 6
2017 4
2018 3
2019 6
2020 2
2021 3
2022 7
2023 8
2024 2

Text availability

Article attribute

Article type

Publication date

Search Results

52 results

Results by year

Filters applied: . Clear all
Page 1
Pompe disease gene therapy.
Byrne BJ, Falk DJ, Pacak CA, Nayak S, Herzog RW, Elder ME, Collins SW, Conlon TJ, Clement N, Cleaver BD, Cloutier DA, Porvasnik SL, Islam S, Elmallah MK, Martin A, Smith BK, Fuller DD, Lawson LA, Mah CS. Byrne BJ, et al. Among authors: smith bk. Hum Mol Genet. 2011 Apr 15;20(R1):R61-8. doi: 10.1093/hmg/ddr174. Epub 2011 Apr 25. Hum Mol Genet. 2011. PMID: 21518733 Free PMC article. Review.
Adeno-associated virus-mediated gene therapy for metabolic myopathy.
Mah CS, Soustek MS, Todd AG, McCall A, Smith BK, Corti M, Falk DJ, Byrne BJ. Mah CS, et al. Among authors: smith bk. Hum Gene Ther. 2013 Nov;24(11):928-36. doi: 10.1089/hum.2013.2514. Hum Gene Ther. 2013. PMID: 24164240 Free PMC article. Review.
Respiratory assessment in centronuclear myopathies.
Smith BK, Goddard M, Childers MK. Smith BK, et al. Muscle Nerve. 2014 Sep;50(3):315-26. doi: 10.1002/mus.24249. Epub 2014 Aug 5. Muscle Nerve. 2014. PMID: 24668768 Free PMC article. Review.
The respiratory neuromuscular system in Pompe disease.
Fuller DD, ElMallah MK, Smith BK, Corti M, Lawson LA, Falk DJ, Byrne BJ. Fuller DD, et al. Among authors: smith bk. Respir Physiol Neurobiol. 2013 Nov 1;189(2):241-9. doi: 10.1016/j.resp.2013.06.007. Epub 2013 Jun 21. Respir Physiol Neurobiol. 2013. PMID: 23797185 Free PMC article. Review.
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Shieh PB, Kuntz NL, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Servais L, Smith BK, Muntoni F, Blaschek A, Foley AR, Saade DN, Neuhaus S, Alfano LN, Beggs AH, Buj-Bello A, Childers MK, Duong T, Graham RJ, Jain M, Coats J, MacBean V, James ES, Lee J, Mavilio F, Miller W, Varfaj F, Murtagh M, Han C, Noursalehi M, Lawlor MW, Prasad S, Rico S. Shieh PB, et al. Among authors: smith bk. Lancet Neurol. 2023 Dec;22(12):1125-1139. doi: 10.1016/S1474-4422(23)00313-7. Lancet Neurol. 2023. PMID: 37977713 Free article. Clinical Trial.
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
Lawlor MW, Schoser B, Margeta M, Sewry CA, Jones KA, Shieh PB, Kuntz NL, Smith BK, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Blaschek A, Neuhaus S, Foley AR, Saade DN, Tsuchiya E, Qasim UR, Beatka M, Prom MJ, Ott E, Danielson S, Krakau P, Kumar SN, Meng H, Vanden Avond M, Wells C, Gordish-Dressman H, Beggs AH, Christensen S, Conner E, James ES, Lee J, Sadhu C, Miller W, Sepulveda B, Varfaj F, Prasad S, Rico S. Lawlor MW, et al. Among authors: smith bk. EBioMedicine. 2024 Jan;99:104894. doi: 10.1016/j.ebiom.2023.104894. Epub 2023 Dec 12. EBioMedicine. 2024. PMID: 38086156 Free PMC article.
52 results