While amyloid plaques form first, new work suggests targeting tau tangles that spread through the brain could also help the fight against this devastating disease.
People with erythropoietic protoporphyria (EPP) must go to great lengths to avoid sun exposure. There have been no approved treatments that address the disease mechanism, but a new drug could change that.
A new class of small-molecule drugs could represent a therapeutic modality aimed at eliminating disease-causing proteins before they spread in the body.
A groundbreaking approach targets muscle stem cells to restore regeneration in Duchenne muscular dystrophy, offering new hope beyond traditional dystrophin-focused therapies.