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1319 articles, sorted by publication time.

Gene Therapy · us

Retinal Schisis Closure Maintained for 18 Months: ATSN-201 Gene Therapy Shows Early Signs of Sustained Efficacy

In a small trial involving nine adults, foveal schisis remained closed in seven treated eyes, with some eyes also showing improvements in light sensitivity and visual acuity. These findings offer a potential treatment lead for X-linked retinoschisis, but whether the benefits can consistently extend to more patients awaits confirmation in the ongoing Phase 3 trial.

Drugs and Clinical Research · us

FDA Expands Camzyos Approval: A New Option for Children Weighing at Least 30 Kilograms With Obstructive Hypertrophic Cardiomyopathy

A trial involving 44 adolescents has brought a drug originally used in adults to regulate heart muscle contraction into pediatric care. The study showed reduced obstruction to blood flow out of the heart, but long-term benefits and safety require further follow-up, and the heart failure warning and treatment monitoring requirements remain in place.

Gene and Cell Therapy · uk

Progress reported in gene therapy for inherited retinal disease: VISTA trial improves low-light vision in some patients

Being able to read a few more lines in dim light may represent a meaningful change for people whose vision is gradually deteriorating. The pivotal trial of laru-zova met its primary efficacy endpoint, but how long the improvement lasts and how to weigh the surgical risks remain important questions on the path to approval.

Cancer Research · global

Neoadjuvant Immunotherapy for Melanoma: Five-Year Survival of About 98% in Patients With a Major Pathological Response

Tumor tissue removed during surgery may offer clues for subsequent treatment. An analysis across 26 centers shows outstanding long-term survival among patients who respond well to neoadjuvant immunotherapy and suggests a research direction for reducing postoperative medication; however, prospective trials are still needed to confirm who can safely receive less treatment.