Variant Ataxia Telangiectasia: A Novel ATM Gene Mutation With Disabling Tremor and Response to Deep Brain Stimulation
Objective: We describe a patient with a novel ATM gene variant presenting with tremor, neuropathy, and late-onset ataxia. Background: Ataxia Telangiectasia (AT) is an autosomal…Suvecaltamide Metabolites Are CaV3 Modulators and Contribute to Pharmacological Effect
Objective: To determine the potency-concentration relationships and combined pharmacological effects for suvecaltamide and its 2 active metabolites (JZZ05000034=M01, JZZ05000035=M02; total active moiety [TAM]). Background: T-type…SAGE-324/BIIB124, an investigational GABAA receptor PAM, for treatment of essential tremor
Objective: KINETIC2 is a multicenter, randomized, placebo-controlled, dose-response Ph2b study evaluating SAGE-324/BIIB124 for treatment of essential tremor (ET). Background: ET is a common movement disorder1,2…Contribution to Efficacy by Active Metabolites of Suvecaltamide in a Preclinical Rat Model of Essential Tremor
Objective: Present the pharmacokinetic/pharmacodynamic (PK/PD) relationship and contribution to anti-tremor efficacy for suvecaltamide and its active metabolites (JZZ05000034=M01, JZZ05000035=M02) in a harmaline-induced rat model of…Once-Daily Suvecaltamide Pharmacokinetics, Safety, & Tolerability–Phase 1, Randomized, Double-Blind, Multiple Ascending Dose Study
Objective: Evaluate the pharmacokinetics (PK), safety, and tolerability of a once-daily (QD) suvecaltamide (JZP385) formulation and its major active metabolites (JZZ05000034=M01, JZZ05000035=M02) in a Phase…The Fazekas scale score and the occurrence of adverse events after MRgFUS thalamotomy, is there a relationship?
Objective: To analyse whether the score on the Fazekas scale (FS) correlates with an increased risk of adverse effects (AEs) after unilateral magnetic resonance-guided focused…Essential3: An Innovative Multi-Study Phase 3 Program to Evaluate the Efficacy and Safety of Ulixacaltamide
Objective: An innovative, multi-study Phase 3 program to definitively assess safety and efficacy of ulixacaltamide in essential tremor (ET). Background: Phase 2 studies of ulixacaltamide,…Magnetic resonance-guided focused ultrasounds (MRgFUS) thalamotomy for disabling tremor secondary to multiple sclerosis.
Objective: To describe two patients with disabling tremor secondary to multiple sclerosis (MS) treated with magnetic resonance-guided focused ultrasounds (MRgFUS) thalamotomy. Background: MRgFUS thalamotomy improves…Istradefylline effects on L-Dopa resistant tremor in Parkinson’s disease
Objective: Quantifying Istradefylline(IST) effects in L-Dopa(LD) resistant tremor in Parkinson’s disease(PD) patients. Background: The adenosine A2A receptors control the indirect pathway. Adenosine A2A receptor activation decreases…Assessing the Economic Impact of Transcutaneous Afferent Patterned Stimulation Therapy for Essential Tremor: A Real-World Evidence Study Protocol
Objective: To assess differences in healthcare resource utilization (HCRU) and costs between patients with essential tremor (ET) receiving transcutaneous afferent patterned stimulation (TAPS) therapy and…
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