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Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease affecting motor neurons. In their Review, Shaw and colleagues provide a comprehensive picture of the various pathological mechanisms involved in this complex disease, and discuss the deep and diverse pipeline under development to tackle these processes. They highlight advances in ALS translational research that might be broadly applicable to other neurodegenerative disorders.
Single-agent therapies targeting specific dysregulated pathways in cancer can be highly effective, but drug resistance frequently develops. Here, Bernards and colleagues discuss the mechanisms underlying resistance to targeted therapies, and assess how these can be suppressed by using tailored combination therapies.
Biogen and Denali’s pivotal trial of a first-in-class LRRK2 kinase inhibitor is a long-awaited test of a target that could provide a path to disease-modifying therapies for Parkinson disease.
Our Future Health CEO Andrew Roddam discusses the UK’s largest-ever health research programme, an ambitious attempt to tackle prevention, detection and treatment of common diseases.
Artificial intelligence tools are increasingly being applied in the development and use of medicines. Here, we provide a regulatory perspective on such applications.