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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Detalles Bibliográficos
Publicado en: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: John Wiley and Sons Inc. 2018
Assuntos:
Review‐themed Issue
Acceso en liña: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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