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...

Descripción completa

Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2018
Materias:
Review‐themed Issue
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6533500/
https://www.ncbi.nlm.nih.gov/pubmed/30281842
http://dx.doi.org/10.1111/bcp.13777

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6533500/
https://www.ncbi.nlm.nih.gov/pubmed/30281842
http://dx.doi.org/10.1111/bcp.13777

Ejemplares similares