Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy

por Montes, Jacqueline, Dunaway Young, Sally, Mazzone, Elena S., Pasternak, Amy, Glanzman, Allan M., Finkel, Richard S., Darras, Basil T., Muntoni, Francesco, Mercuri, Eugenio, De Vivo, Darryl C., Bishop, Kathie M., Schneider, Eugene, Bennett, C. Frank, Foster, Richard, Farwell, Wildon
Publicado 2019

IONIS-PKK(Rx) a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production

por Ferrone, Jason D., Bhattacharjee, Gourab, Revenko, Alexey S., Zanardi, Thomas A., Warren, Marshelle S., Derosier, Frederick J., Viney, Nicholas J., Pham, Nguyen C., Kaeser, Gwendolyn E., Baker, Brenda F., Schneider, Eugene, Hughes, Steven G., Monia, Brett P., MacLeod, A. Robert
Publicado 2019

Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies

por Darras, Basil T., Chiriboga, Claudia A., Iannaccone, Susan T., Swoboda, Kathryn J., Montes, Jacqueline, Mignon, Laurence, Xia, Shuting, Bennett, C. Frank, Bishop, Kathie M., Shefner, Jeremy M., Green, Allison M., Sun, Peng, Bhan, Ishir, Gheuens, Sarah, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C.
Publicado 2019

Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data

por Viney, Nicholas J., Guo, Shuling, Tai, Li‐Jung, Baker, Brenda F., Aghajan, Mariam, Jung, Shiangtung W., Yu, Rosie Z., Booten, Sheri, Murray, Heather, Machemer, Todd, Burel, Sebastien, Murray, Sue, Buchele, Gustavo, Tsimikas, Sotirios, Schneider, Eugene, Geary, Richard S., Benson, Merrill D., Monia, Brett P.
Publicado 2020

Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen

por Coelho, Teresa, Waddington Cruz, Márcia, Chao, Chi-Chao, Parman, Yeşim, Wixner, Jonas, Weiler, Markus, Barroso, Fabio A., Dasgupta, Noel R., Jung, Shiangtung W., Schneider, Eugene, Viney, Nicholas J., Dyck, P. James B., Ando, Yukio, Gillmore, Julian D., Khella, Sami, Gertz, Morie A., Obici, Laura, Berk, John L.
Publicado 2022

Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants

por Jones, Simon A., Valayannopoulos, Vassili, Schneider, Eugene, Eckert, Stephen, Banikazemi, Maryam, Bialer, Martin, Cederbaum, Stephen, Chan, Alicia, Dhawan, Anil, Di Rocco, Maja, Domm, Jennifer, Enns, Gregory M., Finegold, David, Gargus, J. Jay, Guardamagna, Ornella, Hendriksz, Christian, Mahmoud, Iman G., Raiman, Julian, Selim, Laila A., Whitley, Chester B., Zaki, Osama, Quinn, Anthony G.
Publicado 2016

Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L(Rx) (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneu...

por Coelho, Teresa, Ando, Yukio, Benson, Merrill D., Berk, John L., Waddington-Cruz, Márcia, Dyck, Peter J., Gillmore, Julian D., Khella, Sami L., Litchy, William J., Obici, Laura, Monteiro, Cecilia, Tai, Li-Jung, Viney, Nicholas J., Buchele, Gustavo, Brambatti, Michela, Jung, Shiangtung W., St. L. O’Dea, Louis, Tsimikas, Sotirios, Schneider, Eugene, Geary, Richard S., Monia, Brett P., Gertz, Morie
Publicado 2021