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Long-Term Evolocumab in Patients With Established Atherosclerotic Cardiovascular Disease.
O'Donoghue ML, Giugliano RP, Wiviott SD, Atar D, Keech A, Kuder JF, Im K, Murphy SA, Flores-Arredondo JH, López JAG, Elliott-Davey M, Wang B, Monsalvo ML, Abbasi S, Sabatine MS. O'Donoghue ML, et al. Circulation. 2022 Oct 11;146(15):1109-1119. doi: 10.1161/CIRCULATIONAHA.122.061620. Epub 2022 Aug 29. Circulation. 2022. PMID: 36031810 Clinical Trial.
Incidences of serious adverse events, muscle-related events, new-onset diabetes, hemorrhagic stroke, and neurocognitive events with evolocumab long term did not exceed those for placebo-treated patients during the parent study and did not increase over time. ...CONC …
Incidences of serious adverse events, muscle-related events, new-onset diabetes, hemorrhagic stroke, and neurocognitive events with evolocum …
Emerging therapies for Duchenne muscular dystrophy.
Markati T, Oskoui M, Farrar MA, Duong T, Goemans N, Servais L. Markati T, et al. Lancet Neurol. 2022 Sep;21(9):814-829. doi: 10.1016/S1474-4422(22)00125-9. Epub 2022 Jul 15. Lancet Neurol. 2022. PMID: 35850122 Review.
However, glucocorticoids remain the only treatment unequivocally shown to slow disease progression, despite the adverse effects associated with their long-term use. Besides glucocorticoids, which are standard care, five compounds have received regulatory approval in some b …
However, glucocorticoids remain the only treatment unequivocally shown to slow disease progression, despite the adverse effects associated w …
Association Between Achieved Low-Density Lipoprotein Cholesterol Levels and Long-Term Cardiovascular and Safety Outcomes: An Analysis of FOURIER-OLE.
Gaba P, O'Donoghue ML, Park JG, Wiviott SD, Atar D, Kuder JF, Im K, Murphy SA, De Ferrari GM, Gaciong ZA, Toth K, Gouni-Berthold I, Lopez-Miranda J, Schiele F, Mach F, Flores-Arredondo JH, López JAG, Elliott-Davey M, Wang B, Monsalvo ML, Abbasi S, Giugliano RP, Sabatine MS. Gaba P, et al. Circulation. 2023 Apr 18;147(16):1192-1203. doi: 10.1161/CIRCULATIONAHA.122.063399. Epub 2023 Feb 13. Circulation. 2023. PMID: 36779348 Clinical Trial.
In the open-label extension (FOURIER-OLE), 6635 of these patients were transitioned to open-label evolocumab regardless of initial treatment allocation in the parent trial and were followed for an additional median of 5 years. ...CONCLUSIONS: In patients with atherosclerot …
In the open-label extension (FOURIER-OLE), 6635 of these patients were transitioned to open-label evolocumab regardless of initial treatment …
Beta-Thalassemia.
Langer AL. Langer AL. 2000 Sep 28 [updated 2024 Feb 8]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. 2000 Sep 28 [updated 2024 Feb 8]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. PMID: 20301599 Free Books & Documents. Review.
Treatment with regular red blood cell transfusions and iron chelation therapy allows for normal growth and development and improves prognosis. Long-term complications associated with iron overload include stunted growth, dilated cardiomyopathy, liver disease, and endocrino …
Treatment with regular red blood cell transfusions and iron chelation therapy allows for normal growth and development and improves prognosi …
Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 6 Years with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Clinical Trial.
Wainwright C, McColley SA, McNally P, Powers M, Ratjen F, Rayment JH, Retsch-Bogart G, Roesch E, Ahluwalia N, Chin A, Chu C, Lu M, Menon P, Waltz D, Weinstock T, Zelazoski L, Davies JC. Wainwright C, et al. Am J Respir Crit Care Med. 2023 Jul 1;208(1):68-78. doi: 10.1164/rccm.202301-0021OC. Am J Respir Crit Care Med. 2023. PMID: 37154609
Methods: In this phase 3, two-part (part A and part B), open-label extension study, children aged 6 years with CF heterozygous for F508del and a minimal function CFTR mutation (F/MF genotypes) or homozygous for F508del (F/F genotype) who completed the 24-week …
Methods: In this phase 3, two-part (part A and part B), open-label extension study, children aged 6 years with CF heterozygous for F508del a …
Trajectories of Growth Associated With Long-Term Stimulant Medication in the Multimodal Treatment Study of Attention-Deficit/Hyperactivity Disorder.
Greenhill LL, Swanson JM, Hechtman L, Waxmonsky J, Arnold LE, Molina BSG, Hinshaw SP, Jensen PS, Abikoff HB, Wigal T, Stehli A, Howard A, Hermanussen M, Hanć T; MTA Cooperative Group. Greenhill LL, et al. J Am Acad Child Adolesc Psychiatry. 2020 Aug;59(8):978-989. doi: 10.1016/j.jaac.2019.06.019. Epub 2019 Aug 15. J Am Acad Child Adolesc Psychiatry. 2020. PMID: 31421233 Free PMC article.
OBJECTIVE: To estimate long-term stimulant treatment associations on standardized height, weight, and body mass index trajectories from childhood to adulthood in the Multimodal Treatment Study of Attention-Deficit/Hyperactivity Disorder (MTA). ...RESULTS: Height z scores t …
OBJECTIVE: To estimate long-term stimulant treatment associations on standardized height, weight, and body mass index trajectories fr …
Long-term follow-up of patients with relapsing multiple sclerosis from the CLARITY/CLARITY Extension cohort of CLASSIC-MS: An ambispective study.
Giovannoni G, Boyko A, Correale J, Edan G, Freedman MS, Montalban X, Rammohan K, Stefoski D, Yamout B, Leist T, Aydemir A, Borsi L, Verdun di Cantogno E. Giovannoni G, et al. Mult Scler. 2023 May;29(6):719-730. doi: 10.1177/13524585231161494. Epub 2023 Apr 3. Mult Scler. 2023. PMID: 37012898 Free PMC article.
BACKGROUND: CLASSIC-MS evaluated the long-term efficacy of cladribine tablets in patients with relapsing multiple sclerosis. OBJECTIVE: Report long-term mobility and disability beyond treatment courses received in CLARITY/CLARITY Extension. ...
BACKGROUND: CLASSIC-MS evaluated the long-term efficacy of cladribine tablets in patients with relapsing multiple sclerosis. OBJECTIV …
Long-term safety and efficacy of ozanimod in relapsing multiple sclerosis: Up to 5 years of follow-up in the DAYBREAK open-label extension trial.
Cree BA, Selmaj KW, Steinman L, Comi G, Bar-Or A, Arnold DL, Hartung HP, Montalbán X, Havrdová EK, Sheffield JK, Minton N, Cheng CY, Silva D, Kappos L, Cohen JA. Cree BA, et al. Mult Scler. 2022 Oct;28(12):1944-1962. doi: 10.1177/13524585221102584. Epub 2022 Jun 28. Mult Scler. 2022. PMID: 35765217 Free PMC article. Clinical Trial.
During DAYBREAK, 2143 patients (85.9%) had treatment-emergent adverse events (TEAEs; similar in nature to those in the parent trials), 298 (11.9%) had a serious TEAE, and 75 (3.0%) discontinued treatment due to TEAEs. ...Adjusted mean numbers of new/enlarging T2 lesions/sc …
During DAYBREAK, 2143 patients (85.9%) had treatment-emergent adverse events (TEAEs; similar in nature to those in the parent trials) …
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.
Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. Patients & methods: Study 019 was a …
Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patien …
Efficacy and safety of evolocumab in reducing lipids and cardiovascular events.
Sabatine MS, Giugliano RP, Wiviott SD, Raal FJ, Blom DJ, Robinson J, Ballantyne CM, Somaratne R, Legg J, Wasserman SM, Scott R, Koren MJ, Stein EA; Open-Label Study of Long-Term Evaluation against LDL Cholesterol (OSLER) Investigators. Sabatine MS, et al. N Engl J Med. 2015 Apr 16;372(16):1500-9. doi: 10.1056/NEJMoa1500858. Epub 2015 Mar 15. N Engl J Med. 2015. PMID: 25773607 Free article. Clinical Trial.
BACKGROUND: Evolocumab, a monoclonal antibody that inhibits proprotein convertase subtilisin-kexin type 9 (PCSK9), significantly reduced low-density lipoprotein (LDL) cholesterol levels in short-term studies. We conducted two extension studies to obtain longer-term …
BACKGROUND: Evolocumab, a monoclonal antibody that inhibits proprotein convertase subtilisin-kexin type 9 (PCSK9), significantly reduced low …
286 results