A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis

Por Waddington Cruz, Márcia, Benson, Merril D.
Publicado no Neurol Ther (2015)

Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy

Por Waddington Cruz, Márcia, Amass, Leslie, Keohane, Denis, Schwartz, Jeffrey, Li, Huihua, Gundapaneni, Balarama
Publicado no Amyloid (2016)

Early intervention with tafamidis provides long-term benefit in delaying neurological progression in patients with transthyretin familial amyloid polyneuropathy

Por Waddington-Cruz, Marcia, Amass, Leslie, Keohane, Denis, Schwartz, Jeffrey, Gundapaneni, Balarama, Li, Hui Hua
Publicado no Orphanet J Rare Dis (2015)

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)

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)

Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial

Por Coelho, Teresa, Maia, Luis F., Martins da Silva, Ana, Waddington Cruz, Marcia, Planté-Bordeneuve, Violaine, Lozeron, Pierre, Suhr, Ole B., Campistol, Josep M., Conceição, Isabel Maria, Schmidt, Hartmut H.-J., Trigo, Pedro, Kelly, Jeffery W., Labaudinière, Richard, Chan, Jason, Packman, Jeff, Wilson, Amy, Grogan, Donna R.
Publicado em 2012

Assessing mNIS+7(Ionis) and International Neurologists’ Proficiency in a FAP Trial

Por Dyck, Peter J., Kincaid, John C., Dyck, P. James B., Chaudhry, Vinay, Goyal, Namita A., Alves, Christina, Salhi, Hayet, Wiesman, Janice F., Labeyrie, Celine, Robinson-Papp, Jessica, Cardoso, Márcio, Laura, Matilde, Ruzhansky, Katherine, Cortese, Andrea, Brannagan, Thomas H., Khoury, Julie, Khella, Sami, Waddington-Cruz, Márcia, Ferreira, João, Wang, Annabel K., Pinto, Marcus V., Ayache, Samar S., Benson, Merrill D., Berk, John L., Coelho, Teresa, Polydefkis, Michael, Gorevic, Peter, Adams, David H., Plante-Bordeneuve, Violaine, Whelan, Carol, Merlini, Giampaolo, Heitner, Stephen, Drachman, Brian M., Conceição, Isabel, Klein, Christopher J., Gertz, Morie A., Ackermann, Elizabeth J., Hughes, Steven. G., Mauermann, Michelle. L., Bergemann, Rito, Lodermeier, Karen A., Davies, Jenny L., Carter, Rickey E., Litchy, William J.
Publicado no Muscle Nerve (2017)