日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Caregiver-reported Patient Experiences with Duchenne Muscular Dystrophy: Qualitative In-trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial

照护者报告的杜氏肌营养不良症患者体验:关键性 EMBARK 试验中 Delandistrogene Moxeparvovec 治疗 1 年后的定性试验内访谈

Audhya, Ivana; Nacson, Alise B; Gooch, Katherine; Basnyat, Bonita; Slota, Christina; Martin, Susan; Murphy, Alex; Lansdall, Claire J; Ciobanu, Teofil; Nascimento, Andres; Veerapandiyan, Aravindhan

The Balancing Act of Paid Work and Caregiving in Duchenne Muscular Dystrophy (DMD): Results from a Cross-sectional Survey

杜氏肌营养不良症(DMD)患者在有偿工作和照护之间如何平衡:一项横断面调查的结果

Dunne, Jessica S; Szabo, Shelagh M; Gooch, Katherine L; Morrison, Conor L; Audhya, Ivana F

Delays and Barriers Related to the Treatment of Hereditary Angioedema Attacks in Italy

意大利遗传性血管性水肿发作治疗中的延误和障碍

Cancian, Mauro; Accardo, Pietro; Arcoleo, Francesco; Giardino, Francesco; Nicola, Stefania; Senter, Riccardo; Triggianese, Paola; Danese, Sherry; Ulloa, Julie; Desai, Vibha; Frade, Joao P; Audhya, Paul K; Zanichelli, Andrea

A privileged ER compartment for posttranslational heteromeric assembly of an ion channel.

内质网中一个特殊的区室,用于离子通道的翻译后异源组装

Kannan Sudharsan, Kasberg William, Ernandez Liliana R, Audhya Anjon, Robertson Gail A

Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy

来自 EMBARK 的照护者总体印象观察:一项评估 Delandistrogene Moxeparvovec 对可活动杜氏肌营养不良症患者的 3 期研究

McDonald, Craig M; Elkins, Jacob S; Dharmarajan, Sai; Gooch, Katherine; Ciobanu, Teofil; Lansdall, Claire J; Murphy, Alexander P; McDougall, Fiona; Mercuri, Eugenio M; Audhya, Ivana

Impact of hereditary angioedema attacks on health-related quality of life and work productivity

遗传性血管性水肿发作对健康相关生活质量和工作效率的影响

O'Connor, Maeve; Busse, Paula J; Craig, Timothy J; Radojicic, Cristine; Wedner, H James; Danese, Sherry; Ulloa, Julie; Desai, Vibha; Andriotti, Tomas; Audhya, Paul K; Christiansen, Sandra

A sensitive and specific assay to characterize plasma kallikrein activity in plasma from patients with hereditary angioedema

一种灵敏且特异的检测方法,用于表征遗传性血管性水肿患者血浆中的激肽释放酶活性。

Lee, Daniel K; Ghannam, Arije; Murugesan, Nivetha; Vincent, Denis; Dona, Micaela; Cohn, Danny M; Adatia, Adil; Smith, Michael D; Audhya, Paul K; Hampton, Sally L; Feener, Edward P

Organizing principles underlying COPII-mediated transport

COPII介导运输的组织原则

Flood, Julia R; Mendina, Caitlin A; Audhya, Anjon

Psychometric evaluation of the PROMIS parent proxy mobility item bank for use in Duchenne muscular dystrophy

对 PROMIS 家长代理移动能力项目库在杜氏肌营养不良症中的应用进行心理测量学评估

Lowes, Linda Pax; Le Reun, Corinne M; Alfano, Lindsay N; Reash, Natalie F; Iammarino, Megan A; Patel, Shivangi; Audhya, Ivana F

Sebetralstat for On-Demand Treatment of Mucosal Hereditary Angioedema Attacks in KONFIDENT-S

KONFIDENT-S 研究中,Sebetralstat 用于按需治疗粘膜遗传性血管性水肿发作。

Bernstein, Jonathan A; Aygören-Pürsün, Emel; Cancian, Mauro; Cohn, Danny M; Craig, Timothy; Grivcheva-Panovska, Vesna; Jordan, Anthony; Lumry, William R; Martinez-Saguer, Inmaculada; Melamed, Isaac; Ohmura, Kazumasa; Peter, Jonny; Riedl, Marc A; Soteres, Daniel F; Staubach, Petra; Stobiecki, Marcin; Chuang, Ya-Hsiu; Smith, Michael D; Yea, Christopher M; Audhya, Paul K; Zanichelli, Andrea; Farkas, Henriette