RRNMF Neuromuscular Journal最新文献

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A Worsening Problem in ALS: Insurance Barriers Between Drug Approvals and Patient Access 渐冻人症日益严重的问题:药物审批与患者就医之间的保险障碍
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.21232
Richard Bedlack, Daragh Heitzman, Jeremy Shefner
{"title":"A Worsening Problem in ALS: Insurance Barriers Between Drug Approvals and Patient Access","authors":"Richard Bedlack, Daragh Heitzman, Jeremy Shefner","doi":"10.17161/rrnmf.v5i1.21232","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.21232","url":null,"abstract":"","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":"6 13","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140083742","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Primary Care, Private Equity, and Profit 初级保健、私募股权和利润
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.21346
Joshua Freeman
{"title":"Primary Care, Private Equity, and Profit","authors":"Joshua Freeman","doi":"10.17161/rrnmf.v5i1.21346","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.21346","url":null,"abstract":"","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":"2 8","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140087581","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Opening Comments for Vol. 5 Issue 1 第 5 卷第 1 期开幕评论
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.21789
R. Barohn
{"title":"Opening Comments for Vol. 5 Issue 1","authors":"R. Barohn","doi":"10.17161/rrnmf.v5i1.21789","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.21789","url":null,"abstract":"","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":" 610","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140092506","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Recurrent Guillian Barre’ Syndrome vs acute onset CIDP - study of 2 cases. 复发性吉利安-巴雷综合征与急性起病的脊髓损伤性脊髓炎--对两个病例的研究。
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.20953
Sankalp Mohan, M. Dhamne
{"title":"Recurrent Guillian Barre’ Syndrome vs acute onset CIDP - study of 2 cases.","authors":"Sankalp Mohan, M. Dhamne","doi":"10.17161/rrnmf.v5i1.20953","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.20953","url":null,"abstract":"The most common acquired immune mediated polyneuropathies are acute inflammatory demyelinating polyradiculoneuropathy(AIDP), a form of Gullian Barre syndrome (GBS) and chronic inflammatory demyelinating polyradiculoneuropathy(CIDP). Sixteen percent of CIDP patients may present acutely like AIDP, developing in less than 8 weeks. Also GBS which is usually monophasic can have recurrences . Distinguishing Acute onset CIDP(A-CIDP) and recurrent AIDP may be difficult in early stages but may be crucial to guide the treatment strategies.","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":"18 17","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140083556","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Rhabdomyolysis and Exercise Intolerance in a 45-Year-Old Man 一名 45 岁男子的横纹肌溶解症和运动不耐受症
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.21026
Humzah Ahmad, Malvika Govil, Salman Bhai, Chunyu Cai
{"title":"Rhabdomyolysis and Exercise Intolerance in a 45-Year-Old Man","authors":"Humzah Ahmad, Malvika Govil, Salman Bhai, Chunyu Cai","doi":"10.17161/rrnmf.v5i1.21026","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.21026","url":null,"abstract":"A 45-year-old man presented to neuromuscular clinic after a first-time episode of non-traumatic rhabdomyolysis after aerobic exercise. Prior to his diagnosis, he had an extensive medical workup to evaluate for elevated transaminases and creatinine, including liver and renal biopsies. On history, the patient confirmed a lifelong history of exercise intolerance. Creatine kinase evaluation revealed an elevated baseline value. Genetic testing disclosed homozygous variants of uncertain significance and required exercise testing and muscle biopsy to identify the underlying etiology. This case demonstrates pitfalls of genetic testing and an approach to identify this form of myopathy.","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":"15 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140083640","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
What Does It Take To Make a Living Wage? 怎样才能挣到 "维持生计的工资"?
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v3i4.18622
Donald Frey
{"title":"What Does It Take To Make a Living Wage?","authors":"Donald Frey","doi":"10.17161/rrnmf.v3i4.18622","DOIUrl":"https://doi.org/10.17161/rrnmf.v3i4.18622","url":null,"abstract":"","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":"10 20","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140090942","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Pattern Recognition of Neuropathy and Neuronopathy 神经病和神经元病的模式识别
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.21510
R. Barohn, M. Dimachkie, Todd D. Levine, D. Saperstein, Jonathan S. Katz
{"title":"Pattern Recognition of Neuropathy and Neuronopathy","authors":"R. Barohn, M. Dimachkie, Todd D. Levine, D. Saperstein, Jonathan S. Katz","doi":"10.17161/rrnmf.v5i1.21510","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.21510","url":null,"abstract":"","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":" 661","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140092589","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Laboratory testing in peripheral nerve disorders 周围神经疾病的实验室检测
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.21509
Todd D. Levine, R. Barohn, David S. Saperstein, Jonathan S. Katz, Mazen M Dimachkie
{"title":"Laboratory testing in peripheral nerve disorders","authors":"Todd D. Levine, R. Barohn, David S. Saperstein, Jonathan S. Katz, Mazen M Dimachkie","doi":"10.17161/rrnmf.v5i1.21509","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.21509","url":null,"abstract":"","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":" July","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140092822","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Survival and multidisciplinary amyotrophic lateral sclerosis clinic care at a United States Veterans Affairs medical center 美国退伍军人事务医疗中心的存活率和多学科肌萎缩侧索硬化症门诊护理
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.20037
Stephen Rostad, Linder Wendt, Mia Poleksic, Bryan Hutchinson-Reuss, Heather Bingham, D. Fattal
{"title":"Survival and multidisciplinary amyotrophic lateral sclerosis clinic care at a United States Veterans Affairs medical center","authors":"Stephen Rostad, Linder Wendt, Mia Poleksic, Bryan Hutchinson-Reuss, Heather Bingham, D. Fattal","doi":"10.17161/rrnmf.v5i1.20037","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.20037","url":null,"abstract":"Introduction/Aims: The purpose of this work was to investigate survival outcomes in patients with amyotrophic lateral sclerosis (ALS) at our Veteran’s Affairs Medical Center multidisciplinary ALS clinic and compare this to relevant data from several European studies. \u0000Methods: Our sample consisted of 56 total Veterans (n=56; 54 males, 2 females) that had been seen between June 24, 2013 and February 1, 2021 at our multidisciplinary ALS clinic. \u0000Results: The median survival time of our Veterans from symptom onset was 40.96 months (95% CI of 32.17, 76.07), and the median survival time from diagnosis was 23.77 months (95% CI of 18.64, 38.58). This was consistent with the literature. Further consistent with the literature is that multidisciplinary clinics, including ours, have survival advantage over general neurology clinics. Analyzing factors that contributed to this survival, we found significant protective effect on survival from Edaravone use (HR = 0.32, p = 0.036). Otherwise, there was no significant effect on survival noted from use of percutaneous endoscopic gastrostomy (PEG), non-invasive ventilation (NIV), or Riluzole. \u0000Conclusion: We found no significant difference in survival rates between our U.S. Veterans in our multidisciplinary ALS clinic and European multidisciplinary ALS clinics, and both are better than general neurology clinics. We also found that Edaravone use may provide some benefit to survival in this patient population.","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":"39 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140082495","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Safety and tolerability of phenylbutyrate in inclusion body myositis 苯丁酸盐治疗包涵体肌炎的安全性和耐受性
RRNMF Neuromuscular Journal Pub Date : 2024-03-01 DOI: 10.17161/rrnmf.v5i1.21356
D. Jabari, A. Heim, A. Ciersdorff, Heather Wilkins, Abdulbaki Agbas, E. Kosa, Suzanne Hunt, M. Pasnoor, M. Dimachkie, R. Barohn
{"title":"Safety and tolerability of phenylbutyrate in inclusion body myositis","authors":"D. Jabari, A. Heim, A. Ciersdorff, Heather Wilkins, Abdulbaki Agbas, E. Kosa, Suzanne Hunt, M. Pasnoor, M. Dimachkie, R. Barohn","doi":"10.17161/rrnmf.v5i1.21356","DOIUrl":"https://doi.org/10.17161/rrnmf.v5i1.21356","url":null,"abstract":"Introduction \u0000Phenylbutyrate (PBA) showed positive effect on the muscle cell model of Inclusion Body Myositis (IBM) by improving lysosomal activity, ameliorating consequences of impaired autophagy, and decreasing vacuolization. This provides rationale to study this medication in patients with IBM. \u0000Objectives \u0000To evaluate the safety and tolerability of phenylbutyrate in IBM, and monitor for any early signal of effectiveness. \u0000Methods \u0000Open-label study of 10 subjects with IBM who received treatment with PBA for 3 months after a 3-month run-in period. The PBA dose was 3 gm twice daily. The primary outcome measure was adverse event reporting. Secondary outcome measures included manual muscle testing, timed up and go test, IBM functional rating scale, and grip strength, along with exploratory biomarkers evaluating the mitochondrial function, stress response, degenerative process, and apoptosis. \u0000Results \u0000Ten subjects completed the study. PBA was well tolerated with no serious adverse events related to it. The most common adverse events were gastrointestinal related and did not require stopping treatment. One of the biomarkers (MitoTracker) showed a statistically significant drop over the treatment period of the study (p-value of 0.02 for the mean change). There were no statistically significant changes in other secondary outcome measures, but the study was limited by a small sample size and short treatment period. \u0000Conclusions \u0000Phenylbutyrate was safe and well tolerated in patients with IBM in this pilot study. The change in the MitoTracker suggests target engagement, but a Phase II study is needed to confirm and study the efficacy of PBA in IBM","PeriodicalId":309700,"journal":{"name":"RRNMF Neuromuscular Journal","volume":"67 15","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140085324","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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