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Science & Research

Assessment of Cannabidiolic Acid’s Neuroprotective Potential in a TDP-43 Transgenic Mouse Model of Amyotrophic Lateral Sclerosis

Cannabinoids originating from plants, such as Δ9-tetrahydrocannabinol (Δ9-THC), cannabinol, and formulations resembling Sativex, have demonstrated neuroprotective properties in various preclinical models of amyotrophic lateral sclerosis (ALS). Nevertheless, the therapeutic potential of lesser-known phytocannabinoids, including cannabidiolic acid (CBDA), has not been extensively investigated. The present investigation examined the neuroprotective capabilities of CBDA, alongside cannabidivarin, cannabidiol (CBD),…

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Peptides, NAD⁺, and MOTS-c: Evidence-Based Insights into Their Roles in Longevity as of 2025

In the domain of longevity research, peptides have garnered significant attention, yet only a subset demonstrates robust mechanistic support derived from empirical investigations. This review synthesizes validated findings from preclinical and clinical studies, while acknowledging constraints such as the paucity of comprehensive human data. BPC-157: Implications for Tissue Repair and Gastrointestinal Barrier Function Extensive preclinical…

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Targeting Aging Pathways with GLP-1 Analogs: From Metabolic Disorders to Healthspan Extension

During the August convening of the Aging Research and Drug Discovery conference in Copenhagen, representatives from Novo Nordisk and Eli Lilly captivated attendees by positing that glucagon-like peptide-1 receptor agonists (GLP-1RAs)—widely recognized for their efficacy in managing obesity and type 2 diabetes—may represent the inaugural class of longevity therapeutics. In addition to their exceptional performance…

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Advancements in Antimalarial Therapy: Phase III Outcomes for a Novel Ganaplacide-Lumefantrine Combination

Recent Phase III trial data from Novartis have demonstrated the efficacy of GanLum, an innovative antimalarial regimen comprising ganaplacide and lumefantrine, representing the first novel therapeutic approach for malaria in over two decades. The multicenter study encompassed 1,688 participants, including both adults and pediatric populations, across 12 nations in Africa. Key findings revealed parasite clearance…

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Regulatory Developments in Gene Therapy for Huntington’s Disease: Perspectives from uniQure’s Engagement with the U.S. Food and Drug Administration

uniQure N.V., a prominent entity in the gene therapy domain focused on developing innovative interventions for individuals with profound medical conditions, recently disclosed outcomes from a pre-Biologics License Application (BLA) interaction with the U.S. Food and Drug Administration (FDA) concerning AMT-130, an experimental adeno-associated virus-based gene therapy targeting Huntington’s disease (HD). This neurodegenerative disorder, characterized…

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The Neuroprotective Potential of Panax ginseng in Neurodegenerative Diseases: Molecular Mechanisms, Therapeutic Evidence, and Translational Perspectives

Neurodegenerative disorders, encompassing Alzheimer’s disease (AD), Parkinson’s disease (PD), amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and Huntington’s disease (HD), constitute a major and escalating public health burden in ageing societies worldwide. These conditions are defined by the progressive degeneration and loss of specific neuronal populations, resulting in profound cognitive decline, motor disability, and behavioural…

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FDA Approves Itvisma®: A Landmark Gene Therapy for SMA

In a pivotal advancement for rare disease treatment, the U.S. Food and Drug Administration (FDA) has approved Itvisma® (onasemnogene abeparvovec-brve), developed by Novartis, as the first and only gene replacement therapy for children aged 2 years and older, teens, and adults with spinal muscular atrophy (SMA). Announced on November 24, 2025, this approval expands access…

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A Historic Milestone in Personalized Genetic Medicine: Israel’s First Individualized RNA Therapy for a Rare Neurodevelopmental Disorder

In a groundbreaking achievement for precision medicine in Israel — and one of the very few such cases worldwide — a multidisciplinary team at Sheba Medical Center has developed and administered a fully personalized RNA-based therapy for an 8-year-old girl named Ayla, who suffers from a rare and devastating GNAO1-related neurodevelopmental disorder. The treatment,…

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Extracellular Vesicles as Mediators of Systemic Toxic Injury Propagation: A Neuroscience and Molecular Biology Perspective

Extracellular vesicles (EVs), particularly exosomes and microvesicles, are a highly conserved intercellular communication system. Under severe toxic stress, EVs can transfer not only physiological signals but also full cellular injury programs to distant tissues — without the toxin itself ever reaching the target cells. Here we review the robust experimental evidence available as of November…

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Exclusive: Dr. Ariel Ionescu on the microRNA-126 Breakthrough That’s Giving New Hope to ALS

Ariel Ionescu, the scientist who co-founded Rinnerva Therapeutics and has spent years developing a truly innovative therapy for amyotrophic lateral sclerosis (ALS), sat down with Nicolae Tomescu. In this exclusive interview for SLA România – first published on NEUROOTS.co – Dr. Ionescu gives the straight answers to the questions that matter most to the ALS…

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