When to decide to enroll a TTR-FAP patient in a Clinical Trial?

Por Buades, Juan
Publicado no Orphanet J Rare Dis (2015)

Preliminary assessment of neuropathy progression in patients with hereditary ATTR amyloidosis after orthotopic liver transplantation (OLT)

Por Adams, David, Buades, Juan, Suhr, Ole, Obici, Laura, Coelho, Teresa
Publicado no Orphanet J Rare Dis (2015)

Recommendations for presymptomatic genetic testing and management of individuals at risk for hereditary transthyretin amyloidosis

Por Obici, Laura, Kuks, Jan B., Buades, Juan, Adams, David, Suhr, Ole B., Coelho, Teresa, Kyriakides, Theodore
Publicado no Curr Opin Neurol (2016)

Phase 2 open-label extention (OLE) study of patisiran, an investigational siRNA agent for familial amyloidotic polyneuropathy (FAP)

Por Adams, David, Suhr, Ole B, Conceicao, Isabel, Waddington-Cruz, Marcia, Schmidt, Hartmut, Buades, Juan, Campistol, Josep, Coehlo, Teresa
Publicado no Orphanet J Rare Dis (2015)

Cardiac extracellular volume quantified with T1 mapping techniques reflects degree of cardiac and neurological involvement in Hereditary Transthyretin Amyloidosis

Por González-López, Esther, Gallego-Delgado, María, Muñoz-Beamud, Francisco, Buades, Juan, Galán, Lucía, Blanco, Jose Luis Muñoz, González-Mirelis, Jesús, García-Pavía, Pablo
Publicado no Orphanet J Rare Dis (2015)

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Por Suhr, Ole B, Coelho, Teresa, Buades, Juan, Pouget, Jean, Conceicao, Isabel, Berk, John, Schmidt, Hartmut, Waddington-Cruz, Márcia, Campistol, Josep M., Bettencourt, Brian R., Vaishnaw, Akshay, Gollob, Jared, Adams, David
Publicado no Orphanet J Rare Dis (2015)