Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP)

Clinical trials for orphan diseases are critical for developing effective therapies. One such condition, fibrodysplasia ossificans progressiva (FOP; MIM#135100), is characterized by progressive heterotopic ossification (HO) that leads to severe disability. Individuals with FOP are extremely sensitiv...

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Detalhes bibliográficos
Publicado no:Br J Clin Pharmacol
Main Authors: Hsiao, Edward C., Di Rocco, Maja, Cali, Amanda, Zasloff, Michael, Al Mukaddam, Mona, Pignolo, Robert J., Grunwald, Zvi, Netelenbos, Coen, Keen, Richard, Baujat, Genevieve, Brown, Matthew A., Cho, Tae‐Joon, De Cunto, Carmen, Delai, Patricia, Haga, Nobuhiko, Morhart, Rolf, Scott, Christiaan, Zhang, Keqin, Diecidue, Robert J., Friedman, Clive S., Kaplan, Fredrick S., Eekhoff, Elisabeth M.W.
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2018
Assuntos:
Review‐themed Issue
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6533500/
https://ncbi.nlm.nih.gov/pubmed/30281842
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/bcp.13777
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