Neurology News Network for the week ending July 10, 2021.
This week Neurology News Network covered a newly developed version of the Narcolepsy Severity Scale, the clinical benefit amantadine extended-release capsules have on improving motor aspects of daily living, and the neuroprotective properties demonstrated by pridopidine in patients with Huntington disease and amyotrophic lateral sclerosis.
Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.
A recent assessment has determined that newly developed version of the Narcolepsy Severity Scale, dubbed the pediatric Narcolepsy Severity Scale (NSS-P), appears to be a useful clinical tool associated with self-reported sleepiness, insomnia, and depressive symptoms among school-aged children and adolescents with narcolepsy type 1. All told, the NSS-P demonstrated adequate psychometric properties with significant correlations to the item-total score. Additionally, specific factor analysis indicated that the scale offered a 4-factor solution with good reliability. Those individuals who were treated for NT1 (n = 92) reported lower NSS-P scores than those who were untreated (n = 68), with a mean difference of 3.71 points and a minimal clinically important difference estimated at 4 points. The study authors told NeurologyLive, “This scale constitutes a relevant tool to improve and provide guidance for NT1 management in pediatric populations. The ease of administration, its good psychometric properties, and its sensitivity to detect symptom changes after treatment ensure NSS-P future use in clinical and research settings.”
When treated with amantadine extended-release capsules (Gocovri; Adamas), patients with Parkinson disease saw improvements in both dyskinesia and motor aspects of experiences of daily living (M-EDLs), namely tremor, freezing, eating, and getting in and out of the seated position, according to findings from 2 randomized phase 3 trials published in Neurology Therapy. Changes from baseline to week 12 were analyzed via the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS), with a treatment difference of –2.0 for amantadine versus placebo. Additionally, at the 12-week mark, the amantadine group’s change from baseline exceeded a published minimal clinically important difference threshold of 3.05 points.
A trio of recently published peer-reviewed journal articles have provided insights into pridopidine’s (Prilenia Therapeutics) neuroprotective properties through activation of the sigma-1 receptor (S1P) in neurodegenerative diseases. Pridopidine, a first-in-class small molecule currently in development for the treatment of Huntington disease (HD) and amyotrophic lateral sclerosis (ALS), showed an ability to enhance mitochondrial function and reduce mutant human huntingtin (mHTT)-induced stressed, which are impaired in HD and are mediated by the S1R.Pridopidine is currently also being assessed in the phase 3 PROOF-HD study, which evaluates the safety and efficacy of the drug in 45-mg doses on Total Functional Capacity in patients with early-stage HD.Additionally, the recently completed PRIDE-HD study also showed a maintenance of functional capacity with the treatment compared to placebo in patients with early-stage HD.
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