Výsledky vyhledávání - "M. Dimachkie"

Akademický článek

Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease

Autor: Priya S. Kishnani, Barry J. Byrne, Kristl G. Claeys, Jordi Díaz-Manera, Mazen M. Dimachkie, Hani Kushlaf, Tahseen Mozaffar, Mark Roberts, Benedikt Schoser, Noemi Hummel, Agnieszka Kopiec, Fred Holdbrook, Simon Shohet, Antonio Toscano, on behalf of the PROPEL Study Group

Publikováno v: Journal of Patient-Reported Outcomes, Vol 8, Iss 1, Pp 1-9 (2024)

Abstract Background Late-onset Pompe disease (LOPD), a rare autosomal recessive multisystemic disorder, substantially impacts patients’ day-to-day activities, outcomes, and health-related quality of life (HRQoL). The PROPEL trial compared cipagluco

Externí odkaz: https://doaj.org/article/6c94129535e0439fa3e890c6a89ede54

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Akademický článek

Resistance exercise in early-stage ALS patients, ALSFRS-R, Sickness Impact Profile ALS-19, and muscle transcriptome: a pilot study

Autor: Omar Jawdat, Jason Rucker, Tomoki Nakano, Kotaro Takeno, Jeffery Statland, Mamatha Pasnoor, Mazen M. Dimachkie, Carla Sabus, Yomna Badawi, Suzanne L. Hunt, Naoko H. Tomioka, Sumedha Gunewardena, Clark Bloomer, Heather M. Wilkins, Laura Herbelin, Richard J. Barohn, Hiroshi Nishimune

Publikováno v: Scientific Reports, Vol 14, Iss 1, Pp 1-11 (2024)

Abstract Amyotrophic lateral sclerosis (ALS) patients lack effective treatments to maintain motor and neuromuscular function. This study aimed to evaluate the effect of a home-based exercise program on muscle strength, ALS scores, and transcriptome i

Externí odkaz: https://doaj.org/article/e3237184e1bb4c6f98062c0787594ebf

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Akademický článek

Safety and tolerability of intravenous immunoglobulin in patients with active dermatomyositis: results from the randomised, placebo-controlled ProDERM study

Autor: Rohit Aggarwal, Joachim Schessl, Christina Charles-Schoeman, Zsuzsanna Bata-Csörgő, Mazen M. Dimachkie, Zoltan Griger, Sergey Moiseev, Chester V. Oddis, Elena Schiopu, Jiri Vencovský, Irene Beckmann, Elisabeth Clodi, Todd Levine, the ProDERM investigators

Publikováno v: Arthritis Research & Therapy, Vol 26, Iss 1, Pp 1-13 (2024)

Abstract Background Dermatomyositis is an idiopathic inflammatory myopathy characterised by rashes and progressive muscle weakness. The recent ProDERM (Progress in DERMatomyositis) study is the first large randomised, placebo-controlled trial to esta

Externí odkaz: https://doaj.org/article/c02e04d1eff84292a11f1bc97c530c10

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Akademický článek

Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease

Autor: Matthias Boentert, Kenneth I. Berger, Jordi Díaz-Manera, Mazen M. Dimachkie, Alaa Hamed, Lionel Riou França, Nathan Thibault, Pragya Shukla, Jack Ishak, J. Jaime Caro

Publikováno v: Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-8 (2024)

Abstract Background Clinical trials for rare diseases often include multiple endpoints that capture the effects of treatment on different disease domains. In many rare diseases, the primary endpoint is not standardized across trials. The win ratio ap

Externí odkaz: https://doaj.org/article/e7b737f3c80f41c2a3616eefc9531926

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Akademický článek

Conducting a bayesian multi-armed trial with response adaptive randomization for comparative effectiveness of medications for CSPN

Autor: Alexandra R. Brown, Byron J. Gajewski, Dinesh Pal Mudaranthakam, Mamatha Pasnoor, Mazen M. Dimachkie, Omar Jawdat, Laura Herbelin, Matthew S. Mayo, Richard J. Barohn

Publikováno v: Contemporary Clinical Trials Communications, Vol 36, Iss , Pp 101220- (2023)

Background: Response adaptive randomization is popular in adaptive trial designs, but the literature detailing its execution is lacking. These designs are desirable for patients/stakeholders, particularly in comparative effectiveness research, due to

Externí odkaz: https://doaj.org/article/b52e6d82e8a64ea2a96d8818dd50de39

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Akademický článek

Randomized Trial of Tocilizumab in the Treatment of Refractory Adult Polymyositis and Dermatomyositis

Autor: Chester V. Oddis, Howard E. Rockette, Lei Zhu, Diane C. Koontz, David Lacomis, Swamy Venturupalli, Siamak Moghadam‐Kia, Dana P. Ascherman, Leslie Crofford, Mazen M. Dimachkie, Floranne Ernste, David Gazeley, Galina Marder, Rohit Aggarwal

Publikováno v: ACR Open Rheumatology, Vol 4, Iss 11, Pp 983-990 (2022)

Objective To assess the efficacy and tolerability of tocilizumab in a multicenter, randomized, double‐blind, placebo‐controlled trial in refractory adult patients with dermatomyositis (DM) and polymyositis (PM). Methods Thirty‐six subjects with

Externí odkaz: https://doaj.org/article/3f29f397a65a46d68d5dc727acfb139e

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Akademický článek

Imaging biomarkers in the idiopathic inflammatory myopathies

Autor: Adeel S. Zubair, Sharfaraz Salam, Mazen M. Dimachkie, Pedro M. Machado, Bhaskar Roy

Publikováno v: Frontiers in Neurology, Vol 14 (2023)

Idiopathic inflammatory myopathies (IIMs) are a group of acquired muscle diseases with muscle inflammation, weakness, and other extra-muscular manifestations. IIMs can significantly impact the quality of life, and management of IIMs often requires a

Externí odkaz: https://doaj.org/article/ebc13fc571b84692a4d57ff0a6d14c63

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