A clinical trial showed that Rhapsido can safely and effectively treat chronic spontaneous urticaria; its developer Novartis hopes that’s just the start.
Emerging optogenetic, gene, and cell therapies are moving beyond single-gene treatments, offering hope for a broader range of patients with retinitis pigmentosa.
QDX and Prelude Therapeutics are developing drugs that selectively target the JAK2 V617F mutation, offering hope for patients with myeloproliferative neoplasms.
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.