<scp>GM1</scp> ‐gangliosidosis: The caregivers' assessments of symptom impact and most important symptoms to treat-Reference-Cited by-同舟云学术

GM1 ‐gangliosidosis: The caregivers' assessments of symptom impact and most important symptoms to treat

Published:2022-12-21 Issue:2 Volume:191 Page:408-423
ISSN:1552-4825
Container-title:American Journal of Medical Genetics Part A
language:en
Short-container-title:American J of Med Genetics Pt A

Author:

Bingaman Amanda¹^ORCID,Waggoner Christine²,Andrews Sara M.¹,Pangonis Diana³,Trad Marie⁴,Giugliani Roberto⁵^ORCID,Giorgino Ruben⁶,Jarnes Jeanine⁷,Vakili Rojan⁸,Ballard Victoria⁹,Peay Holly L.¹

Affiliation:

1. RTI International Research Triangle North Carolina United States

2. Cure GM1 Foundation Albany California United States

3. National Tay‐Sachs & Allied Diseases Association (NTSAD) Brighton Massachusetts United States

4. Lysogene Neuilly‐sur‐Seine France

5. PPGBM UFRGS, DASA Genomics and Casa dos Raros Porto Alegre Rio Grande do Sul Brazil

6. Azafaros Leiden Zuid‐Holland The Netherlands

7. Department of Pediatrics University of Minnesota Minneapolis Minnesota United States

8. Cure GM1 Foundation Member Albany CA USA

9. Passage Bio, Inc. Philadelphia Pennsylvania United States

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/ajmg.a.63038

Reference21 articles.

1. Identifying the Benefits and Risks of Emerging Treatments for Idiopathic Pulmonary Fibrosis: A Qualitative Study

2. Valuing citizen and patient preferences in health: recent developments in three types of best–worst scaling

3. Infantile gangliosidoses: Mapping a timeline of clinical changes

4. The juvenile gangliosidoses: A timeline of clinical change

5. The natural history of Type 1 infantile GM1 gangliosidosis: A literature-based meta-analysis

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