Objective: To study the effect of the levorotatory form of 5-hydroxytryptophan on the cerebellar symptoms of Friedreich's ataxia.
Design: Cooperative double-blind study of the levorotatory form of 5-hydroxytryptophan vs placebo.
Setting: Twelve centers in research hospitals.
Patients: Twenty-six patients were included; 19 completed the study (mean +/- SD age of patients, 25.9 +/- 8.1 years). Of these 19 patients, eight were treated with placebo and 11 were treated with the drug.
Main Outcome Measures: A semiquantitative scale for kinetic and static ("postural") cerebellar functions and quantitative measurements of time in standard tests that evaluated stance, speech, writing, and drawing.
Results: In the active treatment group, a significant decrease of the kinetic score was observed (P = .03), indicating an improvement in coordination.
Conclusions: These results demonstrated that the levorotatory form of 5-hydroxytryptophan is able to modify significantly the cerebellar symptoms in patients with Friedreich's ataxia. However, the effect is only partial and not clinically major.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1001/archneur.1995.00540290042016 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
A single-dose, randomized, crossover bioequivalence study of levamlodipine besilate tablets in healthy subjects.
Naunyn Schmiedebergs Arch Pharmacol
October 2024
Phase I Clinical Trial Laboratory, Affiliated Hospital to Changchun University of Chinese Medicine, Changchun, China.
Levamlodipine, the levorotatory form of amlodipine racemate, has a blood pressure-lowering effect that is twice that of the racemate. The study aims to establish a foundation for the clinical application of the test drug by conducting a phase I clinical bioequivalence trial, comparing its bioequivalence and safety with the reference drug in healthy Chinese subjects. Recruiting 26 healthy subjects for separate bioequivalence trials in both fasting and fed conditions.
View Article and Find Full Text PDFChiral Hydroxy Metabolite of Mebendazole: Analytical and Semi-Preparative High-Performance Liquid Chromatography Resolution and Chiroptical Properties.
Pharmaceuticals (Basel)
May 2024
National Centre for the Control and Evaluation of Medicines, Chemical Medicines Unit, Istituto Superiore di Sanità, Viale Regina Elena 299, 00161 Rome, Italy.
Mebendazole () is a benzimidazole carbamate anthelmintic used worldwide for the treatment and prevention of parasitic disorders in animals and humans. A large number of in vivo and in vitro studies have demonstrated that also has anticancer activity in multiple types of cancers. After oral administration, the phenylketone moiety of is rapidly reduced to the hydroxyl group to form the chiral hydroxy metabolite ().
View Article and Find Full Text PDFClinical evidence of interventions assessed in Friedreich ataxia: a systematic review.
Ther Adv Rare Dis
November 2022
PTC Therapeutics, Zug, Switzerland.
Objectives: The rare inherited autosomal recessive disease Friedreich ataxia (FA) causes progressive neurodegenerative changes and disability in patients. A systematic literature review (SLR) was carried out to understand and summarize the published efficacy and safety of therapeutic interventions in this disease.
Methods: Database searches were carried out in MEDLINE, Embase, and Cochrane by two independent reviewers.
Chemoenzymatic Synthesis of Optically Active Ethereal Analog of -Moramide-A Novel Potentially Powerful Analgesic †.
Int J Mol Sci
October 2022
Laboratory of Biocatalysis and Biotransformation, Department of Drugs Technology and Biotechnology, Faculty of Chemistry, Warsaw University of Technology, Koszykowa St. 75, 00-662 Warsaw, Poland.
To develop potent and safer analgesics, we designed and synthesized a novel enantiomerically enriched ethereal analog of (R)-iso-moramide, namely 2-[(2R)-2-(morpholin-4-yl)propoxy]-2,2-diphenyl-1-(pyrrolidin-1-yl)ethan-1-one. The titled active agent can potentially serve as a powerful synthetic opiate with an improved affinity and selectivity toward opioid receptors (ORs). This hypothesis was postulated based on docking studies regarding the respective complexes between the designed ligand and µ-OR, δ-OR, and κ-OR.
View Article and Find Full Text PDFfinDr: A web server for D-peptide ligand identification.
Synth Syst Biotechnol
December 2021
Signalling Research Centres BIOSS and CIBSS, University of Freiburg, Schänzlestr. 18, 79104, Freiburg, Germany.
In the rapidly expanding field of peptide therapeutics, the short half-life of peptides represents a considerable limitation for drug action. D-peptides, consisting entirely of the dextrorotatory enantiomers of naturally occurring levorotatory amino acids (AAs), do not suffer from these shortcomings as they are intrinsically resistant to proteolytic degradation, resulting in a favourable pharmacokinetic profile. To experimentally identify D-peptide binders to interesting therapeutic targets, so-called mirror-image phage display is typically performed, whereby the target is synthesized in D-form and L-peptide binders are screened as in conventional phage display.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!