Niu, Isabella http://orcid.org/0000-0002-7190-079X
Hsiao, Edward C. http://orcid.org/0000-0001-8924-106X
Wustrack, Rosanna http://orcid.org/0000-0001-8512-5344
Wysolmerski, John J. http://orcid.org/0000-0003-3037-1008
Dann, Pamela
Masharani, Umesh http://orcid.org/0000-0002-3269-804X
Funding for this research was provided by:
National Institutes of Health (DK007418-41, 2R01HD076248)
University of California, San Francisco
National Institutes of Health (1R01HD100468)
Ipsen Biopharmaceuticals
Ultragenyx Pharmaceutical
Clementia Pharmaceuticals
University of Pennsylvania MDBR / FD / MAS Alliance
International Fibrodysplasia Ossificans Progressiva Association
Article History
Received: 17 March 2023
Accepted: 18 May 2023
First Online: 26 June 2023
Declarations
:
: ECH receives clinical trials support from Clementia Pharmaceuticals, an Ipsen Company; and Ultragenyx, Inc. UM receives clinical trial support from Clementia Pharmaceuticals. All authors declare that there are no conflicts of interest regarding the publication of this article.
: This retrospective chart review study involving a human participant was in accordance with the ethical standards of the institutional and national research committee and with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards. The study was conducted with approval from the UCSF Committee for Human Research, as part of the UCSF Biospecimens and Skeletal Tissues for Rare and Orphan Disease Genetics (BSTROnG) Biobank, approved by the Human Investigation Committee (IRB) of University of California, San Francisco (UCSF IRB#10-03053). The participant was consented prior to use of specimens or clinical data for research, and has consented to the publication of the case.
: ECH serves in a volunteer capacity on the Medical Registry Advisory Board of the International Fibrodysplasia Ossificans Progressiva Association. ECH also serves in a volunteer capacity on the Fibrous Dysplasia Foundation Medical Advisory Board. ECH is a member of the International Clinical Council on FOP.