Researchupdated Aug 08, 2020
Development of a patient-reported outcome questionnaire for aplastic anemia and paroxysmal nocturnal hemoglobinuria (PRO-AA/PNH)
Kimmo Weisshaar, Hannah Ewald, Jörg P. Halter, Sabine Gerull, Sandra Schönfeld, Yuliya Senft, Maria Martinez, and 8 more authors
Under Review
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Researchupdated Aug 07, 2020
Clinical features of a Chinese female nongestational choriocarcinoma cohort: a retrospective study of 37 patients
Yuming Shao, Yang Xiang, Fang Jiang, Boju Pan, Xirun Wan, Junjun Yang, Fengzhi Feng, and 2 more authors
Under Review
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Researchupdated Aug 06, 2020
A Retrospective Review of the Contribution of Rare Diseases to Paediatric Mortality in Ireland
Emer Anne Gunne, Cliona McGarvey, Karina Hamilton, Eileen Treacy, Deborah Lambert, Sally Ann Lynch
Under Review
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Researchposted Aug 05, 2020
Is Quarantine for COVID-19 Pandemic Associated with Psychological Burden in Primary Ciliary Dyskinesia? A Prospective Study.
Maria Pia Riccio, Melissa Borrelli, Maria Teresa Fioretti, Margherita Del Bene, Carmela Bravaccio, Marco Poeta, Francesca Santamaria
Under Review
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Researchposted Aug 05, 2020
A Comprehensive Follow-Up Study Identifying Diagnostic Evidences for Secondary Finding
shu li, li haoyu, xiao neng, lin yixiang, yu wenxian, li yingjing, xi hui, and 4 more authors
Under Review
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Researchposted Aug 03, 2020
The prognostic value of NLR for early death in secondary Hemophagocytic lymphohistiocytosis patients: an analysis of 92 patients
linhui hu, fan wu, Ying Pan, jiakui zhang, qian li, weiwei zhu, chen ding, and 4 more authors
Under Review
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Researchposted Aug 02, 2020
Quality of Life of Children With Spinal Muscular Atrophy and Their Caregivers: A Chinese Cross-sectional Study
Mei Yao, Ying Ma, Ruiying Qian, Yu Xia, Changzheng Yuan, Guannan Bai, Shanshan Mao
Under Review
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Researchposted Aug 01, 2020
“I have SMA, SMA doesn’t have me”- A Qualitative Snapshot Into the Challenges, Successes, and Quality of Life of Sma Adolescents and Young Adults
Allison Joy Mazzella, Mary Curry, Lisa Belter, Rosangel Cruz, Jill Jarecki
Under Review
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Researchposted Aug 01, 2020
Patient and Family Experience With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) and Polyneuropathy (ATTR-PN) Amyloidosis: Results of Two Focus Groups
David Rintell, Dena Heath, Florencia Braga Menendez, Elizabeth Cross, Theodore Cross, Vincent Knobell, Bruno Gagnon, and 3 more authors
Under Review
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Researchposted Aug 01, 2020
Impact of Spinal Muscular Atrophy on Caregivers’ Daily Activities and Health-Related Quality of Life
Er Chen, Komal Bawa, Josh Noone, Sarah Whitmire, Daniel Buchenberger, William David Arnold, Rosalina Mills, and 1 more authors
Under Review
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