Angelo M Taveira-DaSilva,Joel Moss
Angelo M Taveira-DaSilva
Introduction: Lymphangioleiomyomatosis (LAM) is a disease of women characterized by cystic lung destruction, lymphatic involvement, and renal angiomyolipomas. ...
Treatment of Rapidly Progressive Systemic Sclerosis: Current and Futures Perspectives [0.03%]
系统性硬化症的治疗：现状和展望
Fabian A Mendoza,Maryah Mansoor,Sergio A Jimenez
Fabian A Mendoza
Introduction: Systemic Sclerosis (SSc) is a systemic autoimmune disease characterized by severe and often progressive cutaneous, pulmonary, cardiac and gastrointestinal tract fibrosis, cellular and humoral immunologic alt...
Recombinant adeno-associated virus vectors in the treatment of rare diseases [0.03%]
重组腺相关病毒载体在罕见病治疗中的应用
Eric Hastie,R Jude Samulski
Eric Hastie
Introduction: An estimated 25 million Americans are living with rare diseases. Adeno-associated virus (AAV)-mediated gene therapy is an emerging therapeutic option for the more than 7,000 identified rare diseases. This pa...
Michael C Burns,Aidan O&#x;Donnell,Igor Puzanov
Michael C Burns
Introduction: Since 2010 multiple targeted therapies and immunotherapies have been approved for the treatment of advanced melanoma. Pembrolizumab, a humanized monoclonal antibody directed against programed death receptor ...
Y Shimodaira,E Elimova,R Wadhwa et al.
Y Shimodaira et al.
Introduction: In 2014, the U.S. Food and Drug Administration (FDA) approved ramucirumab for use in the second line setting of advanced or metastatic, gastric or gastroesophageal adenocarcinoma (GEAC) based on the result o...
Novel Therapeutic Strategies for the Management of Ventricular Arrhythmias Associated with the Brugada Syndrome [0.03%]
布鲁加达综合征室性心律失常的治疗策略
Bence Patocskai,Charles Antzelevitch
Bence Patocskai
Introduction: Brugada syndrome (BrS) is an inherited cardiac arrhythmia syndrome characterized by prominent J waves appearing as distinct coved type ST segment elevation in the right precordial leads of the ECG. It is ass...
Genetics and prospective therapeutic targets for Sjögren-Larsson Syndrome [0.03%]
Sjögren-Larsson综合征的遗传学和前瞻性治疗靶点
William B Rizzo
William B Rizzo
Introduction: Sjögren-Larsson syndrome (SLS) is a rare neurocutaneous disease characterized by ichthyosis, spasticity, intellectual disability and a distinctive retinopathy. It is caused by inactivating mutations in ALDH...
Diagnosis, prevalence, and screening of familial dilated cardiomyopathy [0.03%]
家族性扩张型心肌病的诊断、流行病学和筛查方法
Mary Sweet,Matthew R G Taylor,Luisa Mestroni
Mary Sweet
Introduction: Dilated cardiomyopathy (DCM) is the most common cardiomyopathy and occurs often in families. As an inherited disease, understanding the significance of diagnostic procedures and genetic screening within fami...
Evaluating a novel treatment for coronary artery inflammation in acute Kawasaki disease: A Phase I/IIa trial of atorvastatin [0.03%]
评估阿托伐他汀治疗急性川崎病冠状动脉炎的新型疗法：一项I/IIa期临床试验
Adriana H Tremoulet,Sonia Jain,Jane C Burns
Adriana H Tremoulet
Introduction: Since the 1980s, the primary treatment of acute Kawasaki disease (KD) has been intravenous immunoglobulin and aspirin. However, 5-10% of children with acute KD will develop coronary artery abnormalities desp...
Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy [0.03%]
遗传性肌肉萎缩性心肌病的基因治疗前景
Yongping Yue,Ibrahim M Binalsheikh,Stacey B Leach et al.
Yongping Yue et al.
Introduction: Cardiac involvement is a common feature in muscular dystrophies. It presents as heart failure and/or arrhythmia. Traditionally, dystrophic cardiomyopathy is treated with symptom-relieving medications. Identi...