Tofersen: First Approval

Review Biotechnology & Applied Microbiology

Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation

Richard J. Mead et al.

Summary: Amyotrophic lateral sclerosis (ALS) is a devastating disease with degeneration of motor neurons. Despite the challenges, ALS has seen progress in the development of disease-modifying therapies. Significant advancements have been made in ALS research and novel therapeutic approaches are being applied to address unmet medical needs. This review discusses how advanced knowledge and new approaches can lead to effective translation of therapies for ALS and potentially impact drug discovery for neurodegenerative disorders.

NATURE REVIEWS DRUG DISCOVERY (2023)

添加到收藏夹

Article Clinical Neurology

Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study

Michael Benatar et al.

Summary: Despite limited knowledge of the underlying causes, genetic forms of ALS offer a unique opportunity for therapeutic development. The ATLAS study, the first interventional trial in presymptomatic ALS, aims to evaluate the impact of initiating tofersen in presymptomatic carriers of SOD1 variants associated with rapid disease progression. The study has the potential to provide important insights into presymptomatic trials, identification of at-risk individuals, and future treatment paradigms in ALS.

NEUROTHERAPEUTICS (2022)

添加到收藏夹

Article Medicine, General & Internal

Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

Timothy M. Miller et al.

Summary: This study examined the use of tofersen in patients with SOD1 ALS and found that it can reduce protein concentrations in the cerebrospinal fluid and plasma, but does not improve clinical outcomes and is associated with adverse events. Further investigation is needed to evaluate the effects of early initiation of tofersen.

NEW ENGLAND JOURNAL OF MEDICINE (2022)

添加到收藏夹

Review Clinical Neurology

Antisense therapies in neurological diseases

Jean-Baptiste Brunet De Courssou et al.

Summary: Advances in targeted regulation of gene expression have opened up new therapeutic approaches for monogenic neurological diseases, with antisense therapies relying on modified nucleotide sequences showing promise in treating severe and previously untreatable disorders. Despite the success seen in various neurological disorders, there remain many unknowns and challenges to expanding these treatments to other diseases. Further research and development is needed to fully realize the potential of personalized medicine targeting the pathogenic roots of these conditions.

BRAIN (2022)

添加到收藏夹

Article Medicine, General & Internal