Health‐related quality of life in adults with osteogenesis imperfecta

Author:

Murali Chaya N.12ORCID,Slater Brady1,Musaad Salma34,Cuthbertson David5,Nguyen Dianne1,Turner Alicia1,Azamian Mahshid1,Tosi Laura6,Rauch Frank7,Sutton V. Reid12,Lee Brendan12,Nagamani Sandesh C. S.12ORCID,

Affiliation:

1. Department of Molecular and Human Genetics Baylor College of Medicine Houston Texas USA

2. Texas Children's Hospital Houston Texas USA

3. Department of Pediatrics‐Nutrition Baylor College of Medicine Houston Texas USA

4. Children's Nutrition Research Center United States Department of Agriculture/Agricultural Research Service Houston Texas USA

5. College of Medicine University of South Florida Tampa Florida USA

6. Bone Health Program Children's National Health System Washington D.C. Washington USA

7. Shriner's Hospital of Children McGill University Montreal Quebec Canada

Funder

Eunice Kennedy Shriver National Institute of Child Health and Human Development

National Center for Advancing Translational Sciences

National Institutes of Health

Osteogenesis Imperfecta Foundation

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Reference38 articles.

1. US Food and Drug Administration.Guidance for industry: patient‐reported outcome measures: use in medical product development to support labeling claims 2009.https://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/UCM193282.pdf.

2. Patient reported outcome measures in rare diseases: a narrative review

3. Quality of life in rare genetic conditions: A systematic review of the literature

4. US Food and Drug Administration.Rare diseases: common issues in drug development guidance for industry DRAFT guidance 2019.https://www.fda.gov/drugs/guidancecomplianceregulatoryinformation/guidances/default.htm.

5. To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases

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