The Spinal Muscular Atrophy Health Index: A novel outcome for measuring how a patient feels and functions-Reference-Cited by-同舟云学术

The Spinal Muscular Atrophy Health Index: A novel outcome for measuring how a patient feels and functions

Published:2021-03-24 Issue:6 Volume:63 Page:837-844
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle & Nerve

Author:

Zizzi Christine E.¹²,Luebbe Elizabeth¹,Mongiovi Phillip¹,Hunter Michael³,Dilek Nuran¹,Garland Connie⁴,Ciafaloni Emma¹,Zaidman Craig M.⁵,Kissel John T.⁶,McDermott Michael P.¹²⁷,Johnson Nicholas⁸^ORCID,Sansone Valeria⁹,Heatwole Chad R.¹²

Affiliation:

1. Department of Neurology University of Rochester Rochester New York USA

2. Center for Health and Technology Rochester New York USA

3. Department of Neurology University of California Irvine Orange California USA

4. Department of Pediatric Neurological Surgery Indiana University School of Medicine Indianapolis Indiana USA

5. Department of Neurology Washington University in St. Louis School of Medicine St. Louis Missouri USA

6. Department of Neurology The Ohio State University Wexner Medical Center Columbus Ohio USA

7. Department of Biostatistics and Computational Biology University of Rochester Rochester New York USA

8. Department of Neurology Virginia Commonwealth University Richmond Virginia USA

9. NEuroMuscular Omnicentre (NEMO), Piazza Ospedale Maggiore 3 Milan Italy

Publisher

Wiley

Subject

Physiology (medical),Cellular and Molecular Neuroscience,Neurology (clinical),Physiology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mus.27223

Reference14 articles.

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2. Gene for chronic proximal spinal muscular atrophies maps to chromosome 5q

3. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

4. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

5. US Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and Research Center for Biologics Evaluation and Research Center for Devices and Radiological Health. Guidance for industry patient‐reported outcome measures: use in medical product development to support labeling claims. Silver Spring MD: Office of Communications Division of Drug Information FDA. 2009.

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