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Orphazyme Doses First ALS Patient in Phase 3 Trial of Arimoclomol

The first patient has been dosed in a Phase 3 clinical trial (NCT03491462) evaluating the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). One of the disease mechanisms known to be involved in the development of ALS is protein misfolding and aggregation…

Physicians Asked to Help ALS Patients Die Must Evaluate Motivations, Alternatives, Case Report Says

Physicians who help amyotrophic lateral sclerosis (ALS) patients die should carefully evaluate their motivations, capacity, and care goals, while also discussing alternatives with their patient, according to researchers. An ALS patient case and commentary, “How Should Physicians Care for Dying Patients with Amyotrophic Lateral Sclerosis?” appeared in the…

High-tech Devices Improve Quality of Life, Should Be Standard Care in ALS, Review Says

High-tech communication devices, such as eye-tracking computer systems (ETCS), improve quality of life and enable caregiver-independent interaction of severely disabled patients with amyotrophic lateral sclerosis (ALS). However, technical aspects and patients’ cognitive impairment are among the factors still limiting their use. The review study titled “Communication…

Repair Cells Change in Neurodegenerative Disorders, Promoting Muscle Wasting and Scarring, Study Says

Cells that normally help to repair injured muscle tissue — called fibro-adipogenic progenitors — become key players in the muscle wasting and scarring processes in neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), a study has found. Conducted by researchers at Sanford Burnham Prebys Medical Discovery Institute (SBP), in…