The primary purpose of the Japanese myasthenia gravis registry (JAMG-R) has been to research and promote high-quality medical care for MG patients in Japan. We reviewed the findings of surveys performed by JAMG-R over an ~10-y period. The first goal for favorable quality of life (QOL) is a status of minimal manifestations (MM) or better with an oral prednisolone (PSL) dose of 5 mg/d or less (MM-5 mg).Early and aggressive use of nonoral fast-acting treatment together with low-dose oral PSL (the "EFT strategy") is recommended to reduce disease severity with minimal oral steroid use so that the MM-5 mg target can be met as soon as possible. We conducted the fourth largest multicenter survey ever in 2021, obtaining detailed clinical information from 1710 consecutive MG patients all over Japan, and compared the 2021 surveys with those from 2012 and 2015. The frequency of patients treated with EFT strategies showed a gradual increase, reaching 39% of the total MG patients in the 2021 survey. The current and maximum dose of PSL and the number of days at high-dose (>20 mg/d) PSL showed decreases. Survey results indicate that as EFT

Miller Fisher syndrome (MFS) is a variant of Guillain-Barré syndrome. Delayed facial palsy (DFP) is a symptom that occurs after other neurological symptoms begin to recover within four weeks from the onset of MFS. As there have been few detailed reports about DFP in MFS cases treated with additional immunotherapy, we investigated three cases of DFP in MFS treated with additional steroid therapies. The duration of facial palsy in our cases was 12-24 days. No severe adverse effects were observed. Although adverse side effects should be carefully monitored, additional steroid therapy might be a treatment option for MFS-DFP.

Aim: Our study aimed to clarify how long outpatients with chronic diseases such as hypertension continuously fill prescriptions for the same medications as those prescribed initially and how many medications they take over the long term. Methods: Medication records from April 1, 2016 to March 31, 2017 with total days between initial and final dispensation date ≥330 days and total days of medication supplies ≥180 days were extracted from an electronic database in a Japanese community pharmacy chain. The continuity of refilling medications over 1 year (ie, medication fill adherence) was measured using the medication possession ratio (MPR). Results: A total of 34,549 outpatients received long-term medications under the above conditions (4.4% of all patients in the database). Mean age was 66.0±17.4 years; 63.1% were ≥65 years. The mean number of medications prescribed per patient was 3.2±2.3. More than one-fifth of patients (22.6%) were taking ≥5 medications. The mean MPR for patients overall was 93.6±11.2%; 87.2% of patients had an MPR ≥80% but <110%. Amlodipine besylate, an antihypertensive, was the most commonly prescribed drug (n=5,537 patients). Conclusion: Outpatients that received long-term medications with no change in prescription had an MPR >90% for around 3 medications. It can be reasonably assumed that these patients could receive a longer-term medication supply with the partial fills based on a physician’s instruction (“Bunkatu Chozai” in Japanese). This longer-term supply would be similar to a basic prescription refill, but would require a physician’s signature allowing for partial refills rather than a new prescription for each refill.

A 40-year-old male patient was diagnosed with invasive thymoma and myasthenia gravis in 2015. In 2016 and 2017, he experienced myasthenic crises, with an increase in size of invasive thymoma. In 2018, he received chemotherapy for the invasive thymoma. After 2 months, his symptoms rapidly progressed to myasthenic crisis with severe bulbar and respiratory symptoms, despite the significant effect of chemotherapy for the thymoma. High-dose corticosteroid, multiple plasma exchanges, and intravenous immunoglobulin did not improve the symptoms. Thus, eculizumab was administered, resulting in an improvement in his conditions. To our knowledge, this is the first report showing that eculizumab may improve myasthenic crisis with invasive thymoma.

Myotonic dystrophy type 1 (DM1) is a neuromuscular disorder caused by the genomic expansions of CTG repeats, in which RNA-binding proteins, such as muscleblind-like protein, are sequestered in the nucleus, and abnormal splicing is observed in various genes. Although abnormal splicing reportedly occurs in the brains of patients with DM1, it is relation to the central nervous system symptoms is unknown. Several imaging studies have indicated substantial white matter (WM) defects in patients with DM1. Here, we performed RNA-sequencing and analysis of CTG repeat lengths in the frontal lobe of patients with DM1, separating the grey matter (GM) and WM, to investigate the splicing abnormalities in the DM1 brain, especially in the WM. The results demonstrated the number of repeats in the GM tended to be increase, with several genes showing similar levels of splicing abnormalities in the GM and WM, suggesting that the WM defects in DM1 are not only caused by aberrant splicing of GM RNA but also of WM RNA, which could be attributed to abnormal splicing of glial cell RNAs.