Search

Your search keyword '"Nishino I"' showing total 1,626 results
Start Over Author "Nishino I"
1,626 results on '"Nishino I"'

1. P156 Muscle biopsy findings in a large cohort of patients affected by valosin containing protein disease: preliminary analysis of the international multicentric VCP study

Author

Schiava, M., primary, Nishino, I., additional, Inoue, M., additional, Nishimori, Y., additional, Saito, Y., additional, Polvikoski, T., additional, Charlton, R., additional, Parkhurst, Y., additional, Henderson, M., additional, Marini-Bettolo, C., additional, Guglieri, M., additional, Straub, V., additional, Weihl, C., additional, Stojkovic, T., additional, Villar-Quiles, R., additional, Romero, N., additional, Evangelista, T., additional, Pegoraro, E., additional, De Bleecker, J., additional, Monforte, M., additional, Malfatti, E., additional, Souvannanorath, S., additional, Severa, G., additional, Alonso-Jiménez, A., additional, Baets, J., additional, De Ridder, W., additional, De Jonghe, P., additional, Kierdaszuk, B., additional, Claeys, K., additional, Muelas, N., additional, Oldfors, A., additional, Rodolico, C., additional, Quin, C., additional, Dominguez, C., additional, Lain, A Herández, additional, Pál, E., additional, Papadimas, G., additional, Kushlaf, H., additional, Alfano, L., additional, Alonso-Pérez, J., additional, Luo, S., additional, Badrising, U., additional, Bevilacqua, J., additional, Nedkova-Hristova, V., additional, Cetin, H., additional, Gelpi, E., additional, Klotz, S., additional, and Plana, M Olivé, additional

Published
2023
Full Text
View/download PDF

16. P.178 Clinical classification of variants in the valosin containing protein gene associated with multisystem proteinopathy

Author

Manera, J. Díaz, primary, Schiava, M., additional, Ikenaga, C., additional, Stojkovic, T., additional, Nishino, I., additional, Nair, S., additional, Manousakis, G., additional, Quinn, C., additional, Sahenk, Z., additional, Monforte, M., additional, Oldfors, A., additional, Pal, E., additional, Gomez, B. Velez, additional, de Bleecker, J., additional, Farrugia, M., additional, Harms, M., additional, Ralston, S., additional, Fernandez, J. Sotoca, additional, and Bevilacqua, J., additional

Published
2022
Full Text
View/download PDF

20. DMD/BMD - GENETICS

Author

Okubo, M., primary, Noguchi, S., additional, Hayashi, S., additional, Komaki, H., additional, and Nishino, I., additional

Published
2021
Full Text
View/download PDF

21. DISTAL MYOPATHIES

Author

Mori-Yoshimura, M., primary, Yajima, Y., additional, Kimura, A., additional, Segawa, K., additional, Oya, Y., additional, Mizuno, K., additional, Noguchi, S., additional, Nishino, I., additional, and Takahashi, Y., additional

Published
2021
Full Text
View/download PDF

22. LGMD

Author

Saito, Y., primary, Hayashi, S., additional, Noguchi, S., additional, and Nishino, I., additional

Published
2021
Full Text
View/download PDF

23. DISTAL MYOPATHIES

Author

Yoshioka, W., primary, Sonehara, K., additional, Iida, A., additional, Oya, Y., additional, Kurashige, T., additional, Okubo, M., additional, Ogawa, M., additional, Matsuda, F., additional, Higasa, K., additional, Mori-Yoshimura, M., additional, Nakamura, H., additional, Hayashi, S., additional, Okada, Y., additional, Noguchi, S., additional, and Nishino, I., additional

Published
2021
Full Text
View/download PDF

25. FSHD

Author

Matsumura, T., primary, Nakamura, H., additional, Nishino, I., additional, Sasaki-Honda, M., additional, Suzuki, T., additional, and Komaki, H., additional

Published
2021
Full Text
View/download PDF

26. AUTOIMMUNE & INFLAMMATORY NMD

Author

Tanboon, J., primary, Uruha, A., additional, Arahata, Y., additional, Dittmayer, C., additional, Schweizer, L., additional, Goebel, H., additional, Nishino, I., additional, and Stenzel, W., additional

Published
2021
Full Text
View/download PDF

27. FSHD

Author

Tabata, K., primary, Takeshita, E., additional, Komaki, H., additional, Sumitomo, N., additional, Shimizu-Motohashi, Y., additional, Saito, T., additional, Ishiyama, A., additional, Nakagawa, E., additional, Nishino, I., additional, and Sasaki, M., additional

Published
2021
Full Text
View/download PDF

29. Efficacy and safety of Bimagrumab in sporadic inclusion body myositis

Author

Amato, A.A., Hanna, M.G., Machado, P.M., Badrising, U.A., Chinoy, H., Benveniste, O., Karanam, A.K., Wu, M., Tankó, L.B., Schubert-Tennigkeit, A.A., Papanicolaou, D.A., Lloyd, T.E., Needham, M., Liang, C., Reardon, K.A., de Visser, M., Ascherman, D.P., Barohn, R.J., Dimachkie, M.M., Miller, J.A.L., Kissel, J.T., Oskarsson, B., Joyce, N.C., Van den Bergh, P., Baets, J., De Bleecker, J.L., Karam, C., David, W.S., Mirabella, M., Nations, S.P., Jung, H.H., Pegoraro, E., Maggi, L., Rodolico, C., Filosto, M., Shaibani, A.I., Sivakumar, K., Goyal, N.A., Mori-Yoshimura, M., Yamashita, S., Suzuki, N., Aoki, M., Katsuno, M., Morihata, H., Murata, K., Nodera, H., Nishino, I., Romano, C.D., Williams, V.S.L., Vissing, J., Zhang Auberson, L., Amato, A.A., Hanna, M.G., Machado, P.M., Badrising, U.A., Chinoy, H., Benveniste, O., Karanam, A.K., Wu, M., Tankó, L.B., Schubert-Tennigkeit, A.A., Papanicolaou, D.A., Lloyd, T.E., Needham, M., Liang, C., Reardon, K.A., de Visser, M., Ascherman, D.P., Barohn, R.J., Dimachkie, M.M., Miller, J.A.L., Kissel, J.T., Oskarsson, B., Joyce, N.C., Van den Bergh, P., Baets, J., De Bleecker, J.L., Karam, C., David, W.S., Mirabella, M., Nations, S.P., Jung, H.H., Pegoraro, E., Maggi, L., Rodolico, C., Filosto, M., Shaibani, A.I., Sivakumar, K., Goyal, N.A., Mori-Yoshimura, M., Yamashita, S., Suzuki, N., Aoki, M., Katsuno, M., Morihata, H., Murata, K., Nodera, H., Nishino, I., Romano, C.D., Williams, V.S.L., Vissing, J., and Zhang Auberson, L.

Abstract

Objective To assess long-term (2 years) effects of bimagrumab in participants with sporadic inclusion body myositis (sIBM). Methods Participants (aged 36–85 years) who completed the core study (RESILIENT [Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients]) were invited to join an extension study. Individuals continued on the same treatment as in the core study (10 mg/kg, 3 mg/kg, 1 mg/kg bimagrumab or matching placebo administered as IV infusions every 4 weeks). The co–primary outcome measures were 6-minute walk distance (6MWD) and safety. Results Between November 2015 and February 2017, 211 participants entered double-blind placebo-controlled period of the extension study. Mean change in 6MWD from baseline was highly variable across treatment groups, but indicated progressive deterioration from weeks 24–104 in all treatment groups. Overall, 91.0% (n = 142) of participants in the pooled bimagrumab group and 89.1% (n = 49) in the placebo group had ≥1 treatment-emergent adverse event (AE). Falls were slightly higher in the bimagrumab 3 mg/kg group vs 10 mg/kg, 1 mg/kg, and placebo groups (69.2% [n = 36 of 52] vs 56.6% [n = 30 of 53], 58.8% [n = 30 of 51], and 61.8% [n = 34 of 55], respectively). The most frequently reported AEs in the pooled bimagrumab group were diarrhea 14.7% (n = 23), involuntary muscle contractions 9.6% (n = 15), and rash 5.1% (n = 8). Incidence of serious AEs was comparable between the pooled bimagrumab and the placebo group (18.6% [n = 29] vs 14.5% [n = 8], respectively). Conclusion Extended treatment with bimagrumab up to 2 years produced a good safety profile and was well-tolerated, but did not provide clinical benefits in terms of improvement in mobility. The extension study was terminated early due to core study not meeting its primary endpoint.

Published
2021

30. Clinical practice with steroid therapy for Duchenne muscular dystrophy: An expert survey in Asia and Oceania

Author

Takeuchi, F, Nakamura, H, Yonemoto, N, Komaki, H, Rosales, RL, Kornberg, AJ, Bretag, AH, Dejthevaporn, C, Goh, KJ, Jong, Y-J, Kim, D-S, Khadilkar, SV, Shen, D, Wong, KT, Chai, J, Chan, SH-S, Khan, S, Ohnmar, O, Nishino, I, Takeda, S, Nonaka, I, Takeuchi, F, Nakamura, H, Yonemoto, N, Komaki, H, Rosales, RL, Kornberg, AJ, Bretag, AH, Dejthevaporn, C, Goh, KJ, Jong, Y-J, Kim, D-S, Khadilkar, SV, Shen, D, Wong, KT, Chai, J, Chan, SH-S, Khan, S, Ohnmar, O, Nishino, I, Takeda, S, and Nonaka, I

Abstract

BACKGROUND: Several studies on clinical practice for Duchenne muscular dystrophy (DMD) have been conducted in Western countries. However, there have been only a few similar studies in Asia and Oceania. Here, we investigate the steroid therapy-related clinical practice for DMD among the local experts. In 2015, we conducted a DMD expert survey in Asia and Oceania to acquire information regarding patients with DMD and to assess current clinical practice with the cooperation of Asian and Oceanian Myology Centre, a neuromuscular disease research network. RESULTS: We obtained survey responses from 87 out of 148 clinicians (62%) from 13 countries and regions. In China, 1385 DMD patients were followed-up by 5 respondent neurologists, and 84% were between 0 and 9 years of age (15% were 10-19 years, 1% > 19 years). While in Japan, 1032 patients were followed-up by 20 clinicians, and the age distribution was similar between the 3 groups (27% were 0-9 years, 35% were 10-19 years, 38% were >19 years). Most respondent clinicians (91%) were aware of DMD standard of care recommendations. Daily prednisolone/prednisone administration was used most frequently at initiation (N = 45, 64%). Inconsistent opinion on steroid therapy after loss of ambulation and medication for bone protection was observed. CONCLUSIONS: Rare disease research infrastructures have been underdeveloped in many of Asian and Oceanian countries. In this situation, our results show the snapshots of current medical situation and clinical practice in DMD. For further epidemiological studies, expansion of DMD registries is necessary.

Published
2020

34. OMICs AND AI APPROACHES FOR MUSCLE DISEASES

Author

Kabeya, Y., primary, Okubo, M., additional, Yonezawa, S., additional, Nakano, H., additional, Inoue, M., additional, Ogasawara, M., additional, Saito, Y., additional, Tanboon, J., additional, Indrawati, L., additional, Kumutpongpanich, T., additional, Chen, Y., additional, Tokumasu, R., additional, Iwamori, T., additional, Takano, A., additional, and Nishino, I., additional

Published
2020
Full Text
View/download PDF

35. AUTOIMMUNE MYOPATHIES

Author

Chen, Y., primary, Inoue, M., additional, Ogasawara, M., additional, Saito, Y., additional, Indrawati, L., additional, Tanboon, J., additional, Kumutpongpanich, T., additional, Okubo, M., additional, Yohioka, W., additional, Hayashi, S., additional, Noguchi, S., additional, and Nishino, I., additional

Published
2020
Full Text
View/download PDF

36. CONGENITAL MUSCULAR DYSTROPHIES

Author

Munot, P., primary, McCrea, N., additional, Torelli, S., additional, Manzur, A., additional, Sewry, C., additional, Chambers, D., additional, Feng, L., additional, Ala, P., additional, Zaharieva, I., additional, Ragge, N., additional, Roper, H., additional, Marton, T., additional, Cox, P., additional, Milev, M., additional, Sacher, M., additional, Liang, W., additional, Maruyama, S., additional, Nishino, I., additional, Phadke, R., additional, and Muntoni, F., additional

Published
2020
Full Text
View/download PDF

37. NEW GENES IN NEUROMUSCULAR DISEASES

Author

Inoue, M., primary, Iida, A., additional, Watanabe, K., additional, Hosoi, Y., additional, Miyajima, H., additional, Hayashi, S., additional, Inoue, Y., additional, Inoue, T., additional, Noguchi, S., additional, and Nishino, I., additional

Published
2020
Full Text
View/download PDF

38. FSHD / OPMD / MYOTONIC DYSTROPHY

Author

Kurashige, T., primary, Morino, H., additional, Ueno, H., additional, Murao, T., additional, Watanabe, T., additional, Hinoi, T., additional, Nishino, I., additional, Maruyama, H., additional, and Torii, T., additional

Published
2020
Full Text
View/download PDF

40. CONGENITAL MYOPATHIES 2

Author

Ogasawara, M., primary, Ogawa, M., additional, Nonaka, I., additional, Hayashi, S., additional, Noguchi, S., additional, and Nishino, I., additional

Published
2020
Full Text
View/download PDF

43. FSHD / OPMD / MYOTONIC DYSTROPHY

Author

Sikrova, D., primary, Hamanaka, K., additional, Mitsuhashi, S., additional, Masuda, H., additional, Sekiguchi, Y., additional, Sugiyama, A., additional, Shibuya, K., additional, Lemmers, R., additional, Goossens, R., additional, Ogawa, M., additional, Nagao, K., additional, Obuse, C., additional, Noguchi, S., additional, Hayashi, Y., additional, Kuwabara, S., additional, Balog, J., additional, Nishino, I., additional, and van der Maarel, S., additional

Published
2020
Full Text
View/download PDF

45. Myositis with sarcoplasmic inclusions in Nakajo–Nishimura syndrome: a genetic inflammatory myopathy

Author

Ayaki, T., primary, Murata, K., additional, Kanazawa, N., additional, Uruha, A., additional, Ohmura, K., additional, Sugie, K., additional, Kasagi, S., additional, Li, F., additional, Mori, M., additional, Nakajima, R., additional, Sasai, T., additional, Nishino, I., additional, Ueno, S., additional, Urushitani, M., additional, Furukawa, F., additional, Ito, H., additional, and Takahashi, R., additional

Published
2020
Full Text
View/download PDF

47. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial

Author

Hanna, M.G., Badrising, U.A., Benveniste, O., Lloyd, T.E., Needham, M., Chinoy, H., Aoki, M., Machado, P.M., Liang, C., Reardon, K.A., Visser, M. de, Ascherman, D.P., Barohn, R.J., Dimachkie, M.M., Miller, J.A.L., Kissel, J.T., Oskarsson, B., Joyce, N.C., Bergh, P. van den, Baets, J., Bleecker, J.L. de, Karam, C., David, W.S., Mirabella, M., Nations, S.P., Jung, H.H., Pegoraro, E., Maggi, L., Rodolico, C., Filosto, M., Shaibani, A.I., Sivakumar, K., Goyal, N.A., Mori-Yoshimura, M., Yamashita, S., Suzuki, N., Katsuno, M., Murata, K., Nodera, H., Romano, C.D., Williams, V.S.L., Vissing, J., Auberson, L.Z., Wu, M., Vera, A. de, Papanicolaou, D.A., Amato, A.A., Nishino, I., RESILIENT Study Grp, Neurology, ANS - Neuroinfection & -inflammation, UCL - SSS/IONS/NEUR - Clinical Neuroscience, UCL - (SLuc) Service de neurologie, and RESILIENT Study Grp

Subjects
Adult, Male, 0301 basic medicine, medicine.medical_specialty, Myostatin, Guidelines, Population, Guidelines, Antibodies, Monoclonal, Humanized, Placebo, Myositis, Inclusion Body, law.invention, 03 medical and health sciences, 0302 clinical medicine, Double-Blind Method, Randomized controlled trial, law, Internal medicine, medicine, Humans, education, Adverse effect, Bimagrumab, Aged, Aged, 80 and over, education.field_of_study, business.industry, Repeated measures design, Middle Aged, Myostatin, Settore MED/26 - NEUROLOGIA, Treatment Outcome, 030104 developmental biology, Blood chemistry, Tolerability, Female, Human medicine, Neurology (clinical), business, 030217 neurology & neurosurgery
Abstract

Background Inclusion body myositis is an idiopathic inflammatory myopathy and the most common myopathy affecting people older than 50 years. To date, there are no effective drug treatments. We aimed to assess the safety, efficacy, and tolerability of bimagrumab-a fully human monoclonal antibody-in individuals with inclusion body myositis. Methods We did a multicentre, double-blind, placebo-controlled study (RESILIENT) at 38 academic clinical sites in Australia, Europe, Japan, and the USA. Individuals (aged 3685 years) were eligible for the study if they met modified 2010 Medical Research Council criteria for inclusion body myositis. We randomly assigned participants (1:1:1:1) using a blocked randomisation schedule (block size of four) to either bimagrumab (10 mg/kg, 3 mg/kg, or 1 mg/kg) or placebo matched in appearance to bimagrumab, administered as intravenous infusions every 4 weeks for at least 48 weeks. All study participants, the funder, investigators, site personnel, and people doing assessments were masked to treatment assignment. The primary outcome measure was 6-min walking distance (6MWD), which was assessed at week 52 in the primary analysis population and analysed by intention-to-treat principles. We used a multivariate normal repeated measures model to analyse data for 6MWD. Safety was assessed by recording adverse events and by electrocardiography, echocardiography, haematological testing, urinalysis, and blood chemistry. This trial is registered with ClinicalTrials.gov, number NCT01925209; this report represents the final analysis. Findings Between Sept 26, 2013, and Jan 6, 2016, 251 participants were enrolled to the study, of whom 63 were assigned to each bimagrumab group and 62 were allocated to the placebo group. At week 52, 6MWD change from baseline did not differ between any bimagrumab dose and placebo (least squares mean treatment difference for bimagrumab 10 mg/kg group, 17.6 m, SE 14.3, 99% CI -19.6 to 54.8; p=0.22; for 3 mg/kg group, 18.6 m, 14.2, -18.2 to 55.4; p=0.19; and for 1 mg/kg group, 1.3 m, 14.1, -38.0 to 35.4; p=0.93). 63 (100%) participants in each bimagrumab group and 61 (98%) of 62 in the placebo group had at least one adverse event. Falls were the most frequent adverse event (48 [76%] in the bimagrumab 10 mg/kg group, 55 [87%] in the 3 mg/kg group, 54 [86%] in the 1 mg/kg group, and 52 [84%] in the placebo group). The most frequently reported adverse events with bimagrumab were muscle spasms (32 [51%] in the bimagrumab 10 mg/kg group, 43 [68%] in the 3 mg/kg group, 25 [40%] in the 1 mg/kg group, and 13 [21%] in the placebo group) and diarrhoea (33 [52%], 28 [44%], 20 [32%], and 11 [18%], respectively). Adverse events leading to discontinuation were reported in four (6%) participants in each bimagrumab group compared with one (2%) participant in the placebo group. At least one serious adverse event was reported by 21 (33%) participants in the 10 mg/kg group, 11 (17%) in the 3 mg/kg group, 20 (32%) in the 1 mg/kg group, and 20 (32%) in the placebo group. No significant adverse cardiac effects were recorded on electrocardiography or echocardiography. Two deaths were reported during the study, one attributable to subendocardial myocardial infarction (secondary to gastrointestinal bleeding after an intentional overdose of concomitant sedatives and antidepressants) and one attributable to lung adenocarcinoma. Neither death was considered by the investigator to be related to bimagrumab. Interpretation Bimagrumab showed a good safety profile, relative to placebo, in individuals with inclusion body myositis but did not improve 6MWD. The strengths of our study are that, to the best of our knowledge, it is the largest randomised controlled trial done in people with inclusion body myositis, and it provides important natural history data over 12 months. Copyright (C) 2019 Elsevier Ltd. All rights reserved.

Published
2019

49. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial

Author

Hanna, M. G., Badrising, U. A., Benveniste, O., Lloyd, T. E., Needham, M., Chinoy, H., Aoki, M., Machado, P. M., Liang, C., Reardon, K. A., de Visser, M., Ascherman, D. P., Barohn, R. J., Dimachkie, M. M., Miller, J. A. L., Kissel, J. T., Oskarsson, B., Joyce, N. C., Van den Bergh, P., Baets, J., De Bleecker, J. L., Karam, C., David, W. S., Mirabella, M., Nations, S. P., Jung, H. H., Pegoraro, E., Maggi, L., Rodolico, C., Filosto, M., Shaibani, A. I., Sivakumar, K., Goyal, N. A., Mori-Yoshimura, M., Yamashita, S., Suzuki, N., Katsuno, M., Murata, K., Nodera, H., Nishino, I., Romano, C. D., Williams, V. S. L., Vissing, J., Auberson, L. Z., Wu, M., de Vera, A., Papanicolaou, D. A., Amato, A. A., Mirabella M. (ORCID:0000-0002-7783-114X), Hanna, M. G., Badrising, U. A., Benveniste, O., Lloyd, T. E., Needham, M., Chinoy, H., Aoki, M., Machado, P. M., Liang, C., Reardon, K. A., de Visser, M., Ascherman, D. P., Barohn, R. J., Dimachkie, M. M., Miller, J. A. L., Kissel, J. T., Oskarsson, B., Joyce, N. C., Van den Bergh, P., Baets, J., De Bleecker, J. L., Karam, C., David, W. S., Mirabella, M., Nations, S. P., Jung, H. H., Pegoraro, E., Maggi, L., Rodolico, C., Filosto, M., Shaibani, A. I., Sivakumar, K., Goyal, N. A., Mori-Yoshimura, M., Yamashita, S., Suzuki, N., Katsuno, M., Murata, K., Nodera, H., Nishino, I., Romano, C. D., Williams, V. S. L., Vissing, J., Auberson, L. Z., Wu, M., de Vera, A., Papanicolaou, D. A., Amato, A. A., and Mirabella M. (ORCID:0000-0002-7783-114X)

Abstract

Background: Inclusion body myositis is an idiopathic inflammatory myopathy and the most common myopathy affecting people older than 50 years. To date, there are no effective drug treatments. We aimed to assess the safety, efficacy, and tolerability of bimagrumab—a fully human monoclonal antibody—in individuals with inclusion body myositis. Methods: We did a multicentre, double-blind, placebo-controlled study (RESILIENT) at 38 academic clinical sites in Australia, Europe, Japan, and the USA. Individuals (aged 36–85 years) were eligible for the study if they met modified 2010 Medical Research Council criteria for inclusion body myositis. We randomly assigned participants (1:1:1:1) using a blocked randomisation schedule (block size of four) to either bimagrumab (10 mg/kg, 3 mg/kg, or 1 mg/kg) or placebo matched in appearance to bimagrumab, administered as intravenous infusions every 4 weeks for at least 48 weeks. All study participants, the funder, investigators, site personnel, and people doing assessments were masked to treatment assignment. The primary outcome measure was 6-min walking distance (6MWD), which was assessed at week 52 in the primary analysis population and analysed by intention-to-treat principles. We used a multivariate normal repeated measures model to analyse data for 6MWD. Safety was assessed by recording adverse events and by electrocardiography, echocardiography, haematological testing, urinalysis, and blood chemistry. This trial is registered with ClinicalTrials.gov, number NCT01925209; this report represents the final analysis. Findings: Between Sept 26, 2013, and Jan 6, 2016, 251 participants were enrolled to the study, of whom 63 were assigned to each bimagrumab group and 62 were allocated to the placebo group. At week 52, 6MWD change from baseline did not differ between any bimagrumab dose and placebo (least squares mean treatment difference for bimagrumab 10 mg/kg group, 17·6 m, SE 14·3, 99% CI –19·6 to 54·8; p=0·22; for 3 mg/kg group, 18·6

Published
2019

Catalog

Books, media, physical & digital resources
See catalog results