TL;DR
Gene therapy in heart failure shows variable efficacy with some promising outcomes, and safety concerns and inconsistent results highlight the need for larger, multicenter trials with long-term follow-up.
Stem cell therapies for CHD have demonstrated encouraging biologic signals and generally reassuring early safety data, and the collective findings suggest potential translational applicability of regenerative therapy in the management of congenital heart diseases.
The field demonstrates substantial formulation diversity and biological activity, but translation is constrained by fragmented characterization, sparse comparative evidence, and incomplete assessment of biological fate and safety.
Clinical evidence suggests that gene-based therapies in the management of rare diseases can achieve sustained functional benefits, reduce disease-related complications, and lessen dependence on long-term replacement or supportive treatments.
Background Pharmacological cancer risk reduction has received far less investment than therapeutic oncology and only a few agents have entered routine practice. Understanding the predictors of positive outcomes of previous trials and issues faced is critical to future developments. Methods This systematic review (PROSP...
Abstract Background and aims Evidence-based, mechanism-guided therapies are urgently needed for treating monogenic inflammatory bowel disease (mIBD). For such rare diseases, mechanistic insight is essential to guide treatment when conventional clinical trials are often not feasible. We aimed to summarize literature-bas...
Abstract Amyotrophic lateral sclerosis (ALS) is a highly heterogeneous and fatal neurodegenerative disorder, for which clinical management and drug development have long faced formidable challenges. Since the approval of riluzole and edaravone, dozens of promising drug candidates that showed efficacy in preclinical mod...
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