Therapeutic landscape for Batten disease: current treatments and future prospects.

Batten disease (also known as neuronal ceroid lipofuscinoses) constitutes a family of devastating lysosomal storage disorders that collectively represent the most common inherited paediatric neurodegenerative disorders worldwide. Batten disease can result from mutations in 1 of 13 genes. These mutations lead to a group of diseases with loosely overlapping symptoms and pathology.

Implementing Motor Unit Number Index (MUNIX) in a large clinical trial: Real world experience from 27 centres.

Objective: Motor Unit Number Index (MUNIX) is a quantitative neurophysiological method that reflects loss of motor neurons in Amyotrophic Lateral Sclerosis (ALS) in longitudinal studies. It has been utilized in one natural history ALS study and one drug trial (Biogen USA) after training and qualification of raters.

Methods: Prior to testing patients, evaluators had to submit test-retest data of 4 healthy volunteers.

Thromboelastographic changes in patients experiencing an acute ischemic stroke and receiving alteplase.

Background: Thromboelastography is a method of measuring whole-blood coagulation changes and has been used to guide therapy and monitor changes in a variety of disease states. However, few studies have investigated the thromboelastographic changes experienced in a patient who has received alteplase for an acute ischemic stroke. This pilot study sought to describe the effect of alteplase on the thromboelastogram tracings of patients experiencing an acute ischemic stroke.

The computerized self test (CST): an interactive, internet accessible cognitive screening test for dementia.

The computer self test (CST) is an interactive, internet-based instrument designed to assess functional cognitive domains impaired by Alzheimer's disease (AD) and mild cognitive impairment (MCI). This study consisted of 215 total subjects with a mean age of 75.24.

The Self Test: a screening tool for dementia requiring minimal supervision.

Background: The main purpose of this study was to correlate a newly developed, simple, but comprehensive screening test for dementia called the Self Test (ST) with the Mini-mental State Examination (MMSE), and establish the reliability and validity of the ST.

Methods: The ST was administered to 42 consecutive new referrals to the memory clinic at the Cole Neuroscience Center, University of Tennessee Medical Center (UTMC), Knoxville, TN, U.S.