A single nigral injection of human ENGRAILED-1 induces long-lasting behavior benefit in an experimental primate model of Parkinson Disease
Objective: Evaluate the efficacy of a single nigral injection of human ENGRAILED-1 (hEN1) in a recognized chronic MPTP macaque model. Background: EN1 homeoprotein has been…Preclinical Development of a Novel Carbidopa/Levodopa Precision Olfactory Delivery (POD®) Drug-Device Combination Product for the Treatment of OFF Episodes in Parkinson’s Disease
Objective: This series of preclinical studies of novel levodopa and carbidopa/levodopa powder formulations delivered by the POD device to rats and non-human primates (NHP) guided…Safety of Gocovri in Clinical Practice: One-year Post-launch Pharmacovigilance Data
Objective: To assess the real-world safety profile of Gocovri® (amantadine) extended release capsules one year post-launch. Background: Gocovri received FDA approval in August 2017 for…Successful Treatment of Orthostatic Tremor using Perampanel
Objective: Orthostatic tremor is notoriously difficult to treat. To date, there has only been one case report of marked improvement of symptoms using perampanel. We present…Cockayne Syndrome manifesting as late adult onset cerebellar ataxia: expanding the phenotype
Objective: To report an atypical case of Cockayne Syndrome manifesting as pure adult onset cerebellar ataxia. Background: Cockayne Syndrome (CS) is a rare autosomal recessive…The progression rate of sporadic adult-onset cerebellar ataxia : 1-year follow up study
Objective: To assess progression rate of the sporadic adult-onset ataxia (SAOA) and usefulness of well-known evaluation tools. Background: The sporadic adult-onset ataxia (SAOA) is a…The applicability of the Scale for Assessment and Rating of Ataxia (SARA) in toddlers
Objective: In healthy toddlers (2-4 years of age), we aimed to evaluate: 1. the feasibility of SARA performances and 2. the reliability of SARA sub-score…Another mitochondrial disease mimic: Two Case reports of adult riboflavin transporter deficiency (RTD) type 2 with muscle adenosine monophosphate (AMP) deaminase deficiency
Objective: To present two rare cases of RTD type 2 with co-existing AMP deaminase deficiency. Background: RTD, formerly known as Brown-Vialetto-Van Laere syndrome, is a…Huntington Disease-Like 2: A Case Presentation and Review of the Literature
Objective: To present a case of Huntington Disease-Like 2 (HDL2). HDL2 is rare and phenotypically similar to Huntington’s Disease (HD). However the gene, chromosome, and…Progressive Myoclonus Ataxia: An International Database
Objective: Our aim is to create an international database for patients suffering from the rare syndrome of progressive myoclonus ataxia (PMA), containing their clinical, electrophysiological…
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