Ergothioneine-Mediated Neuroprotection of Human iPSC-Derived Dopaminergic Neurons.

Cell death involving oxidative stress and mitochondrial dysfunction is a major cause of dopaminergic neuronal loss in the substantia nigra (SN) of Parkinson's disease patients. Ergothioneine (ET), a natural dietary compound, has been shown to have cytoprotective functions, but neuroprotective actions against PD have not been well established. 6-Hydroxydopamine (6-OHDA) is a widely used neurotoxin to simulate the degeneration of dopaminergic (DA) neurons in Parkinson's disease.

Predicting amyotrophic lateral sclerosis (ALS) progression with machine learning.

Objective: To predict ALS progression with varying observation and prediction window lengths, using machine learning (ML).

Methods: We used demographic, clinical, and laboratory parameters from 5030 patients in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database to model ALS disease progression as fast (at least 1.5 points decline in ALS Functional Rating Scale-Revised (ALSFRS-R) per month) or non-fast, using Extreme Gradient Boosting (XGBoost) and Bayesian Long Short Term Memory (BLSTM).

Describing and characterising variability in ALS disease progression.

Background, Objectives: Decrease in the revised ALS Functional Rating Scale (ALSFRS-R) score is currently the most widely used measure of disease progression. However, it does not sufficiently encompass the heterogeneity of ALS. We describe a measure of variability in ALSFRS-R scores and demonstrate its utility in disease characterization.

What Do Randomized Controlled Trials Inform Us About Potential Disease-Modifying Strategies for Parkinson's Disease?

Research advances have shed new insight into cellular pathways contributing to PD pathogenesis and offer increasingly compelling therapeutic targets. In this review, we made a broad survey of the published literature that report possible disease-modifying effects on PD. While there are many studies that demonstrate benefits for various therapies for PD in animal and human studies, we confined our search to human "randomised controlled trials" and with the key words "neuroprotection" or "disease-modifying".

A Patient with Limbic Encephalitis, Ear Perichondritis, and Episcleritis - An Unusual Presentation of Relapsing Polychondritis.

Relapsing polychondritis (RPC) is a rare autoimmune disease that is characterized by recurrent inflammation and destruction of cartilaginous tissues. Limbic encephalitis is a rare central nervous system manifestation of RPC that has been mentioned in case reports. Recognition of this association, and reliable methods of diagnosis, including the utility of neuroimaging modalities such as positron emission tomography/computed tomography (PET/CT) can be useful in the evaluation of this diagnostic challenge.

Overturning the Paradigm of Spinal Muscular Atrophy as Just a Motor Neuron Disease.

Spinal muscular atrophy is typically characterized as a motor neuron disease. Untreated patients with the most severe form, spinal muscular atrophy type 1, die early with infantile-onset progressive skeletal, bulbar, and respiratory muscle weakness. Such patients are now living longer due to new disease-modifying treatments such as gene replacement therapy (onasemnogene abeparvovec), recently approved by the US Food and Drug Administration, and nusinersen, a central nervous system-directed treatment which was approved by the US Food and Drug Administration three years ago.

Prospective Cohort Study of Nusinersen Treatment in Adults with Spinal Muscular Atrophy.

Background: The impact of nusinersen therapy on outcomes in adults with Spinal Muscular Atrophy (SMA) remains uncertain.

Objective: To demonstrate whether nusinersen therapy, at currently prescribed doses, can stabilize or improve motor function in adults with SMA using existing outcome measures.

Methods: A single-center prospective cohort study of 6 adults with SMA type 3, with inclusion/exclusion criteria intended to optimize the ability to demonstrate change using established outcome measures.

Advanced neuroimaging in Balo's concentric sclerosis: MRI, MRS, DTI, and ASL perfusion imaging over 1 year.

Balo concentric sclerosis (BCS) is a rare, atypical demyelinating disease, which may rapidly progress to become severe and fatal. Advanced neuroimaging has proven helpful for early diagnosis, classification, prognostication, and monitoring of progression in multiple sclerosis, but has not been fully explored in BCS. We present the case of a 27-year-old woman with BCS in whom advanced neuroimaging was used to correlate the evolution of disease with clinical findings over the course of 1 year.

Trainee Responses to Hurricane Harvey: Correlating Volunteerism With Burnout.

Natural disasters take a heavy toll not only on their victims, but also on physicians who suffer vicarious trauma and burnout. New trainees in Houston, from entering PGY1 residents to entering fellows, underwent even more upheaval and stress during Hurricane Harvey. Many responded to calls for volunteer help.

Discussing edaravone with the ALS patient: an ethical framework from a U.S. perspective.

The recent approval of edaravone by the United States Food and Drug Administration has generated a mix of hope tempered by reality. The costs of the drug, both monetarily and with regard to intensity of treatment, are high. The benefits, while modest, will be viewed through a very different lens by individuals depending on their goals of care.

Recurrent Cerebral Hemorrhage in Normal Pregnancy Secondary to Mycotic Pseudoaneurysms Related to Choriocarcinoma.

Background: Choriocarcinoma coexisting with or after normal pregnancy is extremely rare. To our knowledge, our case report is the first time cerebral mycotic pseudoaneurysms from choriocarcinoma have been proven angiographically.

Case Description: A 38-week pregnant 26-year-old woman presented with an acute left frontal hemorrhage.