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
1983 1
1984 1
1985 2
1986 3
1987 3
1988 3
1989 7
1990 3
1991 1
1992 1
1993 2
1994 1
1995 1
1996 2
2001 1
2003 1
2004 2
2007 2
2008 1
2011 2
2012 5
2013 1
2015 2
2016 5
2017 4
2018 2
2019 1
2020 6
2021 5
2022 11
2023 9
2024 13
2025 13

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

107 results

Results by year

Filters applied: . Clear all
Page 1
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
Diaz-Manera J, Kishnani PS, Kushlaf H, Ladha S, Mozaffar T, Straub V, Toscano A, van der Ploeg AT, Berger KI, Clemens PR, Chien YH, Day JW, Illarioshkin S, Roberts M, Attarian S, Borges JL, Bouhour F, Choi YC, Erdem-Ozdamar S, Goker-Alpan O, Kostera-Pruszczyk A, Haack KA, Hug C, Huynh-Ba O, Johnson J, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Diaz-Manera J, et al. Lancet Neurol. 2021 Dec;20(12):1012-1026. doi: 10.1016/S1474-4422(21)00241-6. Lancet Neurol. 2021. PMID: 34800399 Clinical Trial.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Mercuri E, et al. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
RFC1: Motifs and phenotypes.
Delforge V, Tard C, Davion JB, Dujardin K, Wissocq A, Dhaenens CM, Mutez E, Huin V. Delforge V, et al. Rev Neurol (Paris). 2024 May;180(5):393-409. doi: 10.1016/j.neurol.2024.03.006. Epub 2024 Apr 15. Rev Neurol (Paris). 2024. PMID: 38627134 Free article. Review.
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Kishnani PS, et al. JAMA Neurol. 2023 Jun 1;80(6):558-567. doi: 10.1001/jamaneurol.2023.0552. JAMA Neurol. 2023. PMID: 37036722 Free PMC article. Clinical Trial.
Brain atrophy patterns in anti-IgLON5 disease.
Yogeshwar SM, Bartels F, Grüter T, Muñiz-Castrillo S, Picard G, Crijnen YS, Bernard E, Heidbreder A, Zekeridou A, Ringelstein M, Kraft A, Kovac S, Wandinger KP, de Vries JM, Boon AJW, Veenbergen S, Geis C, Penner L, Melzer N, Leypoldt F, Blaabjerg M, Pittock SJ, Gaig C, Sabater L, Santamaria J, Graus F, Dalmau J, Prüss H, Höftberger R, Schreiner B, McKeon A, Lewerenz J, Irani S, Mignot E, Titulaer MJ, Ayzenberg I, Honnorat J, Finke C; IgLON5 Imaging Consortium. Yogeshwar SM, et al. Brain. 2025 Jul 12:awaf256. doi: 10.1093/brain/awaf256. Online ahead of print. Brain. 2025. PMID: 40650880
[Biological markers and metabolic impact of chronic nitrous oxide consumption].
Grzych G, Deheul S, Davion JB, Lemonnier F, Dobbelaere D, Carton L, Kim I, Guichard JC, Girot M, Humbert L, Joly A, Douillard C, Tard C. Grzych G, et al. Ann Biol Clin (Paris). 2022 Jun 30;80(3):209-212. doi: 10.1684/abc.2022.1729. Ann Biol Clin (Paris). 2022. PMID: 35796471 Free article. French. No abstract available.
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial.
Crawford TO, Servais L, Mercuri E, Kölbel H, Kuntz N, Finkel RS, Krueger J, Batley K, Young SD, Marantz JL, Song G, Yao B, Zhao G, Rossello J, Tirucherai GS, Mazzone ES, Butterfield RJ, de la Banda MGG, Seferian AM, Sansone VA, De Waele L, van der Pol WL, Cances C, Pechmann A, Darras BT; SAPPHIRE Study Group. Crawford TO, et al. Lancet Neurol. 2025 Sep;24(9):727-739. doi: 10.1016/S1474-4422(25)00225-X. Lancet Neurol. 2025. PMID: 40818473 Clinical Trial.
Incidence, prevalence and clinical presentation of inflammatory bowel diseases in Northern France: a 30-year population-based study.
Sarter H, Crétin T, Savoye G, Fumery M, Leroyer A, Dauchet L, Paupard T, Coevoet H, Wils P, Richard N, Turck D, Ley D, Gower-Rousseau C; EPIMAD study Group. Sarter H, et al. Lancet Reg Health Eur. 2024 Oct 18;47:101097. doi: 10.1016/j.lanepe.2024.101097. eCollection 2024 Dec. Lancet Reg Health Eur. 2024. PMID: 39478988 Free PMC article.
Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment.
Desguerre I, Barrois R, Audic F, Barnerias C, Chabrol B, Davion JB, Durigneux J, Espil-Taris C, Gomez-Garcia de la Banda M, Guichard M, Isapof A, Nougues MC, Laugel V, Le Goff L, Mercier S, Pervillé A, Richelme C, Thibaud M, Sarret C, Schweitzer C, Testard H, Trommsdorff V, Vanhulle C, Walther-Louvier U, Altuzarra C, Chouchane M, Ropars J, Quijano-Roy S, Cances C. Desguerre I, et al. Orphanet J Rare Dis. 2024 Sep 13;19(1):344. doi: 10.1186/s13023-024-03326-3. Orphanet J Rare Dis. 2024. PMID: 39272200 Free PMC article.
CUREs for high-level Galectin-3 expression.
Charbonneau AA, Reicks EJ, Cambria JF, Inman J, Danley D, Shockley EA, Davion R, Salgado I, Norton EG, Corbett LJ, Hanacek LE, Jensen JG, Kibodeaux MA, Kirkpatrick TK, Rausch KM, Roth SR, West B, Wilson KE, Lawrence CM, Cloninger MJ. Charbonneau AA, et al. Protein Expr Purif. 2024 Sep;221:106516. doi: 10.1016/j.pep.2024.106516. Epub 2024 May 25. Protein Expr Purif. 2024. PMID: 38801985
107 results