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

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

1,618 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint.
Servais L, Strijbos P, Poleur M, Mirea A, Butoianu N, Sansone VA, Vuillerot C, Schara-Schmidt U, Scoto M, Seferian AM, Previtali SC, Tulinius M, Nascimento A, Furlong P, Singh T, Dreghici RD, Goemans N, Mercuri E, Straub V, Ormazabal MG, Braid J, Muntoni F, Tricot A, Annoussamy M, Eggenspieler D. Servais L, et al. Among authors: nascimento a. Sci Rep. 2024 Nov 29;14(1):29681. doi: 10.1038/s41598-024-80177-9. Sci Rep. 2024. PMID: 39613806 Free PMC article.
Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.
McDonald CM, Muntoni F, Penematsa V, Jiang J, Kristensen A, Bibbiani F, Goodwin E, Gordish-Dressman H, Morgenroth L, Werner C, Li J, Able R, Trifillis P, Tulinius M; Study 019 investigators. McDonald CM, et al. J Comp Eff Res. 2022 Feb;11(3):139-155. doi: 10.2217/cer-2021-0196. Epub 2021 Nov 18. J Comp Eff Res. 2022. PMID: 34791888 Free PMC article. Clinical Trial.
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. Mendell JR, et al. Among authors: nascimento a. Nat Med. 2024 Oct 9. doi: 10.1038/s41591-024-03304-z. Online ahead of print. Nat Med. 2024. PMID: 39385046
Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study.
Crawford TO, Day JW, De Vivo DC, Krueger JM, Mercuri E, Nascimento A, Pasternak A, Mazzone ES, Duong T, Song G, Marantz JL, Baver S, Yu D, Liu L, Darras BT. Crawford TO, et al. Among authors: nascimento a. Front Neurol. 2024 Jul 22;15:1419791. doi: 10.3389/fneur.2024.1419791. eCollection 2024. Front Neurol. 2024. PMID: 39105058 Free PMC article.
The recurrent deep intronic pseudoexon-inducing variant COL6A1 c.930+189C>T results in a consistently severe phenotype of COL6-related dystrophy: Towards clinical trial readiness for splice-modulating therapy.
Foley AR, Bolduc V, Guirguis F, Donkervoort S, Hu Y, Orbach R, McCarty RM, Sarathy A, Norato G, Cummings BB, Lek M, Sarkozy A, Butterfield RJ, Kirschner J, Nascimento A, Benito DN, Quijano-Roy S, Stojkovic T, Merlini L, Comi G, Ryan M, McDonald D, Munot P, Yoon G, Leung E, Finanger E, Leach ME, Collins J, Tian C, Mohassel P, Neuhaus SB, Saade D, Cocanougher BT, Chu ML, Scavina M, Grosmann C, Richardson R, Kossak BD, Gospe SM Jr, Bhise V, Taurina G, Lace B, Troncoso M, Shohat M, Shalata A, Chan SHS, Jokela M, Palmio J, Haliloğlu G, Jou C, Gartioux C, Solomon-Degefa H, Freiburg CD, Schiavinato A, Zhou H, Aguti S, Nevo Y, Nishino I, Jimenez-Mallebrera C, Lamandé SR, Allamand V, Gualandi F, Ferlini A, MacArthur DG, Wilton SD, Wagener R, Bertini E, Muntoni F, Bönnemann CG. Foley AR, et al. Among authors: nascimento a. medRxiv [Preprint]. 2024 Mar 29:2024.03.29.24304673. doi: 10.1101/2024.03.29.24304673. medRxiv. 2024. PMID: 38585825 Free PMC article. Preprint.
Comparison Between Nano-Hydroxyapatite/Beta-Tricalcium Phosphate Composite and Autogenous Bone Graft in Bone Regeneration Applications: Biochemical Mechanisms and Morphological Analysis.
Brum IDS, Frigo L, Ribeiro da Silva JF, Ciambarella BT, Nascimento ALR, Pereira MJDS, Elias CN, de Carvalho JJ. Brum IDS, et al. Among authors: nascimento alr. Int J Mol Sci. 2024 Dec 24;26(1):52. doi: 10.3390/ijms26010052. Int J Mol Sci. 2024. PMID: 39795911 Free PMC article.
Clinical impact of healthcare-associated infections in Brazilian ICUs: a multicenter prospective cohort.
Tomazini BM, Besen BAMP, Santos RHN, Nassar AP Jr, Veiga TS, Campos VB, Tokunaga SM, Santos ES, Barbante LG, da Costa Maia R, Kojima FCS, Laranjeira LN, Taniguchi LU, Roepke RML, Franke CA, Sanches LC, Melro LMG, Maia IS, de Souza Dantas VC, Figueiredo RC, de Alencar Filho MS, Irineu VM, Lovato WJ, Zandonai CL, Machado FR, Arns B, Marsola G, Veiga VC, Pereira AJ, Cavalcanti AB; IMPACTO-MR investigators (2019 - 2023); BRICNet. Tomazini BM, et al. Crit Care. 2025 Jan 3;29(1):4. doi: 10.1186/s13054-024-05203-8. Crit Care. 2025. PMID: 39754200 Free PMC article.
1,618 results