Duchenne muscular dystrophy (DMD) is a progressive, genetic (X-linked recessive) neuromuscular disorder caused by mutations to the DMD gene, resulting in the dysfunction or absence of the dystrophin protein. In DMD, muscle regeneration initially depends on the proliferation and differentiation of muscle satellite cells (MuSCs), but their regenerative capacity progressively declines, making repair inefficient and contributing to muscle dysfunction.

Niagen Bioscience Inc.’s proprietary lead small-molecule drug candidate, NB-4168, has been awarded European orphan drug designation and U.S. rare pediatric disease designation for the treatment of ataxia telangiectasia.

Anorexia nervosa (AN) is a complex and severe eating disorder that has long been considered a psychiatric condition driven by distorted body image and maladaptive eating behaviors. No effective therapy is currently available. At a symposium held during the recent FENS Forum in Barcelona, several leading researchers with decades of experience in AN presented findings that further strengthen the growing body of evidence supporting the disorder’s metabolic component.

Researchers from Connecta Therapeutics SL and the Centre for Genomic Regulation (Barcelona Institute of Science and Technology) recently presented preclinical results on CTH-120, a potential first-in-class allosteric modulator of tropomyosin receptor kinase B (TrkB) designed to enhance neuroplasticity and restore neuronal function in fragile X syndrome.

Researchers from United Arab Emirates University presented the preclinical characterization of ST-2657, a dual G9a/H3R modulator, in a preclinical model of autism.

The Alzheimer’s Association International Conference (AAIC) is the world’s biggest dementia conference. And at the AAIC 2026 meeting, there is big buzz around tau. Sunday’s plenary speaker Ryan Watts, CEO of Denali Therapeutics Inc., highlighted tau-lowering agents as being among the most exciting themes of the conference. “At this conference, we’re going to see additional clinical data that may validate [tau as] a second target in Alzheimer’s disease, amyloid being the first,” he told the audience.

Mindbeam AI has leveraged generative AI to accelerate the discovery of novel pain therapeutics predicted to have improved liver safety compared to acetaminophen.