mNIS+7 and lower limb function in inotersen treatment of hereditary transthyretin‐mediated amyloidosis

di Dyck, P. James B., Kincaid, John C., Wiesman, Janice F., Polydefkis, Michael, Litchy, William J., Mauermann, Michelle L., Ackermann, Elizabeth J., Guthrie, Spencer, Pollock, Michael, Jung, Shiangtung W., Baker, Brenda F., Dyck, Peter J.
Pubblicato in Muscle Nerve (2020)

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

di 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.
Pubblicato in ESC Heart Fail (2020)

Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis

di Dyck, P. James B., Coelho, Teresa, Waddington Cruz, Marcia, Brannagan, Thomas H., Khella, Sami, Karam, Chafic, Berk, John L., Polydefkis, Michael J., Kincaid, John C., Wiesman, Janice F., Litchy, William J., Mauermann, Michelle L., Ackermann, Elizabeth J., Baker, Brenda F., Jung, Shiangtung W., Guthrie, Spencer, Pollock, Michael, Dyck, Peter J.
Pubblicato in Muscle Nerve (2020)

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...

di 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
Pubblicato in Neurol Ther (2021)