Recent years witnessed an exciting increase in the number of clinical trials for neuromuscular disorders, in particular for Duchenne Muscular Dystrophy and Spinal Muscle Atrophy. Given the high emotional impact of such developments for devastating diseases with an urgent medical need, it is particularly important to justify human trials on the basis of robust preclinical studies and to avoid a waste of hopes and of funds.This review focuses the discussion on the quality in the conduct clinically-oriented preclinical assessments in rare neuromuscular disease models and on the importance in reporting of preclinical confirmatory studies. Accordingly, it invites scientists, journal publishers and funding agencies to require quality standards to improve translatability of preclinical findings.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.3233/JND-140067 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Unusual swelling of the hand and multiple nodules over the body - beware of mycobacteria.
Acta Orthop Belg
December 2024
Atypical mycobacteria can cause rare and atypical infections of the hand. We report the case of an immunocompetent 46-year-old male initially presenting with thumb felon and progressively developing symptoms of carpal tunnel syndrome, tenosynovitis of multiple fingers and a sporotrichoid lymphocutaneous infection causing chronic cutaneous lesions all over the body. We would like to highlight the diagnostic and therapeutic difficulties of these atypical infections, which mimic other conditions and can cause a lot of morbidity.
View Article and Find Full Text PDFFactors associated with receiving a Functional Disorder diagnostic label: A systematic review.
PLoS One
January 2025
Interdisciplinary Centre Psychopathology and Emotion Regulation, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands.
Objectives: Functional Disorders (FD) are highly prevalent conditions that are diagnosed based on the presence of specific patterns of somatic symptoms. Examples of FDs include Fibromyalgia and Irritable Bowel Syndrome. Many patients who meet the criteria do not receive a formal diagnostic label.
View Article and Find Full Text PDFAn Algorithmic Overview of Advanced Pain Therapies: A Narrative Review.
Curr Pain Headache Rep
January 2025
Department of Pain Medicine, Division of Anesthesiology, Critical Care & Pain Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX, 77030, USA.
Purpose Of Review: Quickly referenceable, streamlined, algorithmic approaches for advanced pain management are lacking for patients, trainees, non-pain specialists, and interventional specialists. This manuscript aims to address this gap by proposing a comprehensive, evidence-based algorithm for managing neuropathic, nociceptive, and cancer-associated pain. Such an algorithm is crucial for pain medicine education, offering a structured approach for patient care refractory to conservative management.
View Article and Find Full Text PDFDo patients with fibromyalgia syndrome and healthy people differ in their opinions on placebo effects in routine medical care?
Pain Pract
February 2025
Health, Medical and Neuropsychology Unit, Faculty of Social and Behavioural Sciences, Leiden University, Leiden, The Netherlands.
Objectives: Placebo effects can relieve acute and chronic pain in both research and clinical treatments by learning mechanisms. However, the application of placebo-based treatment strategies in routine medical care is questioned. The current study investigated the opinions of patients with fibromyalgia and healthy controls regarding learning of placebo effects and their practical applications.
View Article and Find Full Text PDFA novel muscle network approach for objective assessment and profiling of bulbar involvement in ALS.
Front Neurosci
January 2025
Neurology Associate P.C., Lincoln, NE, United States.
Introduction: As a hallmark feature of amyotrophic lateral sclerosis (ALS), bulbar involvement significantly impacts psychosocial, emotional, and physical health. A validated objective marker is however lacking to characterize and phenotype bulbar involvement, positing a major barrier to early detection, progress monitoring, and tailored care. This study aimed to bridge this gap by constructing a multiplex functional mandibular muscle network to provide a novel objective measurement tool of bulbar involvement.
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!