Reviews & Analysis

A first-in-class therapeutic that targets neuroactive microbial metabolites in the gut shows promising target engagement, safety and behavioral improvements in adolescents with autism spectrum disorder.

A ‘gene addition’ strategy shows promise in a first-in-human trial involving two infant patients.

Studies have demonstrated the value of genomic analysis for the diagnosis of rare diseases, but accessibility is still in its infancy; global data sharing is needed to further advance our knowledge of all causes of rare disease.

Two studies highlight the evolution of antisense oligonucleotide therapy for amyotrophic lateral sclerosis, offering hope for an effective treatment.

Long-term follow-up data reinforce the curative potential of hematopoietic stem-cell gene therapy for this rare primary immunodeficiency disorder.

AI may represent a new tool in the risk assessment and screening pathway of breast cancer. To realise its potential, the effect of AI-supported decisions on relevant clinical outcomes must be prospectively evaluated.