Hemophilia B is an inherited bleeding disorder caused by mutations in the F9 gene, which encodes coagulation factor IX (FIX). Gene therapy, which restores the production of functional FIX by delivering a functional F9 gene, is currently the only potentially curative approach for hemophilia B. Gene therapy products that use liver-targeted adeno-associated virus (AAV) vectors to deliver a highly active FIX variant, FIX-Padua, have demonstrated promising efficacy in clinical trials. To date, two such products have been approved internationally [1–2]. Among them, BBM-H901 (DalnacogenePonparvovec), developed by Professor Xiao Xiao’s team at Belief BioMed, is the first AAV-based gene therapy product for hemophilia B developed in Asia.In 2019, Professor Lei Zhang’s team at our hospital conducted the first clinical study of BBM-H901 in patients with hemophilia B, providing preliminary evidence of its efficacy and safety in Chinese patients. The findings were published in The Lancet Haem...

Source: National Clinical Research Center for Cardiovascular Diseases · Beijing · 6 August 2026 Originally published by the Structural Heart Disease Center, Fuwai Hospital Original URL: https://mp.weixin.qq.com/s/bntJx7B8uEdyWbw-iJwK5A From a "Treatment Dilemma" to a "China Solution" — The COMPETE trial by Professor Pan Xiangbin's team at Fuwai Hospital is published in the top-tier journal The British Medical Journal, exploring a new treatment strategy for migraine associated with patent foramen ovale Migraine is a long-standing complaint that many people cannot shake off. That sudden, severe headache — accompanied by nausea, sensitivity to light and sensitivity to sound — often makes it impossible to work or live normally. Statistically, about 1 billion people worldwide are affected by migraine, and it is the third most common disease in the world. What many people may not know is that inside the bodies of many migraine patients there is an easily overlooked "chan...

On July 8, 2026, the results of the 2025 National Science and Technology Awards were announced. The project titled Establishment and Promotion of a Precision Surgical Treatment System for Severe Heart Failure, led by Academician Hu Shengshou of Fuwai Hospital, Chinese Academy of Medical Sciences, received the Second Prize of the National Science and Technology Progress Award. After nearly two decades of systematic research, the project overcame multiple critical technological bottlenecks, established a comprehensive surgical diagnosis and treatment system for severe heart failure with distinct Chinese characteristics, and advanced China’s capacity for treating severe heart failure into the front ranks internationally. Severe heart failure is a life-threatening end-stage cardiovascular condition with an acute-phase mortality rate as high as 70 percent, a five-year survival rate of only 20 percent, and an extremely poor prognosis. Heart transplantation and implantation of ventri...

Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disease and an important cause of sudden cardiac death in youth. Although HCM is considered to be caused mainly by the pathogenic varants in genes encoding sarcomeric proteins, most commonly MYH7 and MYBPC3, more than half of patients were genetic-negative in the well-characterized disease genes. The considerable genetic heterogeneity of HCM underscores the need to investigate novel pathogenic mechanisms and therapeutic targets, particularly those shared by different causes. Recently, the teams of Prof. Lei Song and Prof. Shuiyun Wang at the National Clinical Research Center for Cardiovascular Diseases, Fuwai Hospital, Chinese Academy of Medical Sciences, in collaboration with Prof. Yihan Chen of Shanghai East Hospital, Tongji University, published a study entitled CRLF1 Secreted by Cardiac Fibroblasts Promotes Human Hypertrophic Cardiomyopathy in Circulation. The study reveals, for the first time, that c...

I. Title The BMJ | Efficacy of dual antiplatelet therapy for three months versus 12 months after coronary artery bypass grafting: multicentre, double blinded, randomised controlled trial. II. Key Findings The BMJ has published online a large, multicenter, randomized, double-blind, controlled clinical trial from China, "TOP-CABG," which demonstrates that in patients undergoing elective coronary artery bypass grafting (CABG), 3 months of dual antiplatelet therapy (DAPT) with ticagrelor plus aspirin is non-inferior to 12-month DAPT in terms of the 1-year saphenous vein graft occlusion rate, and significantly reduces the risk of clinically relevant bleeding. This study provides high-level evidence for an individualized antithrombotic strategy after CABG. Original Publication: https://www.bmj.com/content/393/bmj-2025-088939 III. Main Text Saphenous vein graft occlusion is the major bottleneck limiting the long-term efficacy of coronary artery bypass grafting (CABG). ...

Supported by a series of high-quality clinical studies, sodium–glucose cotransporter 2 inhibitors (SGLT2i) have demonstrated well-established long-term cardiovascular and renal protective effects and play an important role in the management of chronic conditions, including heart failure, diabetes, chronic kidney disease, and chronic cardiovascular disease. However, the efficacy and safety of short-term SGLT2i use during the perioperative period of cardiac surgery remain unclear. SGLT2i exert long-term protective effects through multiple pathways, including the regulation of inflammatory responses, oxidative stress, cellular energy metabolism, systemic metabolic homeostasis, and autonomic function. These pleiotropic mechanisms may provide potential organ protection during the perioperative period of cardiac surgery. However, existing evidence primarily comes from studies of long-term interventions in chronic disease settings, and evidence remains lacking as to whether SGLT2i ca...