Recent clinical data highlight advances in treatments for Alzheimer’s and Duchenne muscular dystrophy. Remynd NV reported positive Phase IIa trial results for its REM-127 candidate in Alzheimer's, demonstrating reductions in pathological tau and cognitive improvement. Simultaneously, Avidity Biosciences released early and midstage data showing disease progression reversal in Duchenne muscular dystrophy patients treated with del-zota. These developments signal therapeutic potential in challenging conditions with high unmet needs, progressing novel mechanisms and modalities towards clinical application.