AI Article Synopsis
- In 1993, the gene linked to familial amyotrophic lateral sclerosis (ALS) due to SOD1 mutations was identified, leading to over 30 years of research into ALS's causes and treatments.
- Tofersen, a promising gene-targeted therapy for ALS, has received approval from the FDA and EMA, but further studies are needed to ensure its safety and effectiveness.
- The paper discusses the current clinical and commercial state of Tofersen and outlines the expectations for its approval in Japan.
Article Abstract
Since the identification, in 1993, of the causative gene for familial amyotrophic lateral sclerosis (ALS), which is associated with SOD1 mutations, research has focused on the pathogenesis and therapeutics of ALS for more than 30 years. Tofersen, a highly anticipated gene-specific therapy that has been aligned with the disease-specific pathology, has been approved for marketing by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) However, as significant data on tofersen's safety and efficacy are required, the evaluation of this treatment is ongoing. This paper introduces the current clinical and commercial status of Tofersen, along with expectations for its approval in Japan.
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| http://dx.doi.org/10.11477/mf.1416202765 | DOI Listing |
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