Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies
Autosomal recessive cerebellar ataxias are disabling neurodegenerative genetic conditions affecting balance and coordination. Advancements in genomic testing have improved diagnosis, leading to a new focus on the development of targeted precision therapeutics addressing cellular, biochemical, and genetic disease mechanisms with a resulting emphasis on ...
Brent L. Fogel+10 more
wiley +1 more source
The effect of octreotide on gastric emptying at a dosage used to prevent complications after pancreatic surgery: a randomised, placebo controlled study in volunteers [PDF]
Mark I. van Berge Henegouwen+4 more
openalex +1 more source
Objective Spinal cord (SC) atrophy correlates with and predicts the underlying progressive biology in active and non‐active multiple sclerosis (MS), thereby providing a biomarker for clinical trials and patient management. Initiation of disease‐modifying therapy (DMT) may be followed by early pronounced central nervous system (CNS) volume loss due to ...
Simone Sacco+26 more
wiley +1 more source
The placebo effect shortens movement time in goal-directed movements. [PDF]
Fiorio M+3 more
europepmc +1 more source
A double‐blind, placebo controlled study of the effect of the specific histamine H1‐receptor antagonist, terfenadine, in chronic severe asthma. [PDF]
Richard Wood‐Baker+2 more
openalex +1 more source
Exenatide Once Weekly in the Treatment of Patients with Multiple System Atrophy
Objective Exenatide, a glucagon‐like peptide‐1 (GLP‐1) receptor agonist, has neuroprotective effects in preclinical models of multiple system atrophy (MSA). We investigated these effects in a proof‐of‐concept clinical trial. Methods In this single‐center, randomized, open label trial, participants with MSA were randomly assigned (1:1) to receive ...
Nirosen Vijiaratnam+29 more
wiley +1 more source
MRI Quantification of Placebo Effect in Nonalcoholic Steatohepatitis Clinical Trials. [PDF]
Nedrud MA+11 more
europepmc +1 more source
Effect of bromazepam versus placebo on inhibition and waiting capacity in young women with traits of anxiety [PDF]
Stéphane Schück+5 more
openalex +1 more source