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Table representation of search results timeline featuring number of search results per year.
Year | Number of Results |
---|---|
2016 | 2 |
2020 | 1 |
2021 | 1 |
2024 | 0 |
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4 results
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Page 1
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
JAMA Neurol. 2021 Jul 1;78(7):834-841. doi: 10.1001/jamaneurol.2021.1272.
JAMA Neurol. 2021.
PMID: 33999158
Free PMC article.
Clinical Trial.
IMPORTANCE: This ongoing study assesses long-term safety and durability of response in infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene abeparvovec gene replacement therapy. ...CONCLUSIONS AND RELEVANCE: The findings of this ongoing clinical …
IMPORTANCE: This ongoing study assesses long-term safety and durability of response in infants with spinal muscular atrophy (SMA) typ …
Clinical Follow-Up for Duchenne Muscular Dystrophy Newborn Screening: A Proposal.
Kwon JM, Abdel-Hamid HZ, Al-Zaidy SA, Mendell JR, Kennedy A, Kinnett K, Cwik VA, Street N, Bolen J, Day JW, Connolly AM.
Kwon JM, et al. Among authors: al zaidy sa.
Muscle Nerve. 2016 Aug;54(2):186-91. doi: 10.1002/mus.25185. Epub 2016 Jun 13.
Muscle Nerve. 2016.
PMID: 27170260
Free PMC article.
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Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.
Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, Shao J, Kaye EM, Mercuri E; Eteplirsen Study Group and Telethon Foundation DMD Italian Network.
Mendell JR, et al.
Ann Neurol. 2016 Feb;79(2):257-71. doi: 10.1002/ana.24555. Epub 2016 Jan 8.
Ann Neurol. 2016.
PMID: 26573217
Free PMC article.
Clinical Trial.
OBJECTIVE: To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD). ...
OBJECTIVE: To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer design …
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Duchenne and Becker muscular dystrophy carriers: Evidence of cardiomyopathy by exercise and cardiac MRI testing.
Mah ML, Cripe L, Slawinski MK, Al-Zaidy SA, Camino E, Lehman KJ, Jackson JL, Iammarino M, Miller N, Mendell JR, Hor KN.
Mah ML, et al. Among authors: al zaidy sa.
Int J Cardiol. 2020 Oct 1;316:257-265. doi: 10.1016/j.ijcard.2020.05.052. Epub 2020 May 27.
Int J Cardiol. 2020.
PMID: 32473283
Clinical Trial Registration https://clinicaltrials.gov/ct2/show/NCT02972580?term=mendell&cond=Duchenne+Muscular+Dystrophy&rank=5; ClinicalTrials.gov Identifier: NCT02972580....
Clinical Trial Registration https://clinicaltrials.gov/ct2/show/NCT02972580?term=mendell&cond=Duchenne+Muscular+Dystrophy&ran …
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