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Lipid nanoparticles and transcranial focused ultrasound enhance the delivery of SOD1 antisense oligonucleotides to the murine brain for ALS therapy.
J Control Release
December 2024
School of Chemistry and Molecular Bioscience, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia. Electronic address:
Article Synopsis
- ALS is a severe neurodegenerative disease characterized by the buildup of misfolded proteins in motor neurons, prompting researchers to find ways to reduce this burden for potential treatment.
- Antisense oligonucleotides (ASOs) have been identified as a promising option to target proteins like SOD1 that cause mutations, but their delivery to the central nervous system is challenging due to the blood-brain barrier.
- The study demonstrates that using transcranial focused ultrasound (FUS) along with calcium phosphate lipid nanoparticles significantly enhances the delivery of a SOD1 ASO into the brain of mice, leading to reduced SOD1 levels and improved motor neuron survival without damaging brain tissue.
Barriers to Tofersen Therapy for Variant SOD1-Mediated ALS.
JAMA Neurol
December 2024
Houston Methodist Neurological Institute, Houston Methodist Research Institute, Stanley H. Appel Department of Neurology, Houston Methodist Hospital, Houston, Texas.
Changes in Cerebrospinal Fluid Concentrations of Selenium Species Induced by Tofersen Administration in Subjects with Amyotrophic Lateral Sclerosis Carrying SOD1 Gene Mutations.
Biol Trace Elem Res
July 2024
Center for Neurosciences and Neurotechnology, Department of Biomedical, Metabolic, and Neural Sciences, University of Modena and Reggio Emilia, Modena, Italy.
Article Synopsis
- ALS is a serious and fatal disease affecting motor neurons, and the FDA recently approved tofersen for treating adults with SOD1 gene mutations.
- A study measured selenium levels in the cerebrospinal fluid of ALS patients before and after tofersen treatment, using advanced mass spectrometry techniques.
- Results showed that tofersen significantly increased selenium concentrations, suggesting it may impact the brain's redox status, indicating a need for further research on the drug's effects and disease progression.
Breaking barriers with tofersen: Enhancing therapeutic opportunities in amyotrophic lateral sclerosis.
Eur J Neurol
February 2024
Department of Pharmaceutical Analysis, ISF College of Pharmacy, Moga, Punjab, India.
Article Synopsis
- Amyotrophic lateral sclerosis (ALS) is a serious neurodegenerative disease that primarily affects muscle control due to the death of motor neurons, and current treatments mainly offer symptomatic relief.
- Tofersen, recently approved by the FDA as Qalsody, is the first gene therapy for ALS that targets a specific genetic cause of the disease linked to mutant SOD1 proteins.
- A thorough literature review shows that with new drugs like tofersen, there's promising progress in monitoring and potentially slowing ALS's progression.
Managed care considerations to improve health care utilization for patients with ALS.
Am J Manag Care
June 2023
President, W-Squared Group, in Longboat Key, FL. Email:
Amyotrophic lateral sclerosis (ALS) is a fatally progressive degenerative disease, with many patients succumbing to the condition within 3 to 5 years after diagnosis. It is a rare, orphan disease with an estimated US prevalence of 25,000 patients. Patients with ALS and their caregivers are faced with a substantial financial burden as a result of the condition, as ALS has an estimated national financial burden of $1.
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