New pharmacotherapies for genetic neuromuscular disorders: opportunities and challenges
Sign Up to like & getrecommendations! Published in 2019 at "Expert Review of Clinical Pharmacology"
DOI: 10.1080/17512433.2019.1634543
Abstract: ABSTRACT Introduction: Genetic neuromuscular diseases (NMDs) constitute a heterogeneous group of rare conditions, including some of the most disabling conditions in childhood. Recently, advanced technologies have greatly expanded preclinical and clinical research, and specific therapies… read more here.
Keywords: neuromuscular disorders; pharmacotherapies genetic; opportunities challenges; disorders opportunities ... See more keywords
Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back
Sign Up to like & getrecommendations! Published in 2020 at "Journal of Personalized Medicine"
DOI: 10.3390/jpm10040258
Abstract: Neuromuscular disorders are a large group of rare pathologies characterised by skeletal muscle atrophy and weakness, with the common involvement of respiratory and/or cardiac muscles. These diseases lead to life-long motor deficiencies and specific organ… read more here.
Keywords: efficacy; clinical trials; men genetic; raav men ... See more keywords