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

62 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
Choice of compound, dosage, and management of side effects for long-term corticosteroid treatment in Duchenne muscular dystrophy: Guidelines from the Neuromuscular Commission of the French Society of Pediatric Neurology.
Fontaine Carbonnel S, Dabaj I, de Montferrand C, Rippert P, Laugel V, De Lucia S, Ravelli C, Seferian A, Ropars J, Cances C. Fontaine Carbonnel S, et al. Among authors: de lucia s. Arch Pediatr. 2024 Oct;31(7):410-418. doi: 10.1016/j.arcped.2024.05.003. Epub 2024 Sep 27. Arch Pediatr. 2024. PMID: 39332946
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
T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy.
Anthony K, Ala P, Catapano F, Meng J, Domingos J, Perry M, Ricotti V, Maresh K, Phillips LC, Servais L, Seferian AM, De Lucia S, de Groot I, Krom YD, Verschuuren JGM, Niks EH, Straub V, Guglieri M, Voit T, Morgan J, Muntoni F. Anthony K, et al. Among authors: de groot i, de lucia s. Hum Gene Ther. 2023 May;34(9-10):439-448. doi: 10.1089/hum.2022.166. Epub 2023 Feb 24. Hum Gene Ther. 2023. PMID: 36453228
Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy.
Fouarge E, Monseur A, Boulanger B, Annoussamy M, Seferian AM, De Lucia S, Lilien C, Thielemans L, Paradis K, Cowling BS, Freitag C, Carlin BP, Servais L; NatHis-MTM Study Group. Fouarge E, et al. Among authors: de lucia s. Orphanet J Rare Dis. 2021 Jan 6;16(1):3. doi: 10.1186/s13023-020-01663-7. Orphanet J Rare Dis. 2021. PMID: 33407688 Free PMC article.
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
Mercuri E, Muntoni F, Baranello G, Masson R, Boespflug-Tanguy O, Bruno C, Corti S, Daron A, Deconinck N, Servais L, Straub V, Ouyang H, Chand D, Tauscher-Wisniewski S, Mendonca N, Lavrov A; STR1VE-EU study group. Mercuri E, et al. Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9. Lancet Neurol. 2021. PMID: 34536405 Clinical Trial.
Genotype-related respiratory progression in Duchenne muscular dystrophy-A multicenter international study.
Trucco F, Ridout D, Domingos J, Maresh K, Chesshyre M, Munot P, Sarkozy A, Robb S, Quinlivan R, Riley M, Wallis C, Chan E, Abel F, De Lucia S, Hogrel JY, Niks EH, de Groot I, Servais L, Straub V, Ricotti V, Manzur A, Muntoni F; UK NorthStar Clinical Network and AFM Network. Trucco F, et al. Among authors: de groot i, de lucia s. Muscle Nerve. 2022 Jan;65(1):67-74. doi: 10.1002/mus.27427. Epub 2021 Oct 27. Muscle Nerve. 2022. PMID: 34606104
North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up.
Coratti G, Pane M, Brogna C, Ricotti V, Messina S, D'Amico A, Bruno C, Vita G, Berardinelli A, Mazzone E, Magri F, Ricci F, Mongini T, Battini R, Bello L, Pegoraro E, Baranello G, Previtali SC, Politano L, Comi GP, Sansone VA, Donati A, Hogrel JY, Straub V, De Lucia S, Niks E, Servais L, De Groot I, Chesshyre M, Bertini E, Goemans N, Muntoni F, Mercuri E; on behalf on the International DMD Group and the iMDEX Consortium. Coratti G, et al. Among authors: de groot i, de lucia s. PLoS One. 2021 Jun 25;16(6):e0253882. doi: 10.1371/journal.pone.0253882. eCollection 2021. PLoS One. 2021. PMID: 34170974 Free PMC article.
Ketogenic diet therapies in France: State of the use in 2018.
Dozières-Puyravel B, François L, de Lucia S, Goujon E, Danse M, Auvin S. Dozières-Puyravel B, et al. Among authors: de lucia s. Epilepsy Behav. 2018 Sep;86:204-206. doi: 10.1016/j.yebeh.2018.05.031. Epub 2018 Jul 8. Epilepsy Behav. 2018. PMID: 29997039
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Servais L, Xiong H, Zanoteli E, Baranello G, Bruno C, Day JW, Deconinck N, Klein A, Mercuri E, Vlodavets D, Wang Y, Dodman A, El-Khairi M, Gorni K, Jaber B, Kletzl H, Gaki E, Fontoura P, Darras BT; FIREFISH Study Group. Masson R, et al. Lancet Neurol. 2022 Dec;21(12):1110-1119. doi: 10.1016/S1474-4422(22)00339-8. Epub 2022 Oct 14. Lancet Neurol. 2022. PMID: 36244364 Clinical Trial.
62 results