Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial von Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.

Volltext

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial von Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Wigderson, Melissa, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.

Volltext

Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study von Crawford, Thomas O, Day, John W, De Vivo, Darryl C, Krueger, Jena M, Mercuri, Eugenio, Nascimento, Andres, Pasternak, Amy, Mazzone, Elena Stacy, Duong, Tina, Song, Guochen, Marantz, Jing L, Baver, Scott, Yu, Dongzi, Liu, Lan, Darras, Basil T

Volltext

Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care von Henderson, Marian L, Zieba, Jacob K, Li, Xiaopeng, Campbell, Daniel B, Williams, Michael R, Vogt, Daniel L, Bupp, Caleb P, Edgerly, Yvonne M, Rajasekaran, Surender, Hartog, Nicholas L, Prokop, Jeremy W, Krueger, Jena M

Volltext

A treatable metabolic cause of encephalopathy: cobalamin C deficiency in an 8-year-old male von Krueger, Jena M, Piantino, Juan, Smith, Craig M, Angle, Brad, Venkatesan, Charu, Wainwright, Mark S

Volltext

Epilepsy Following Neonatal Seizures Secondary to Hemorrhagic Stroke in Term Neonates von Venkatesan, Charu, Millichap, John J., Krueger, Jena M., Nangia, Srishti, Ritacco, David G., Stack, Cynthia, Nordli, Douglas R.

Volltext

Prognosis in Pediatric Myasthenia Gravis von Krueger, Jena

Volltext

Incidence of Dravet Syndrome in a US Population von Krueger, Jena, Berg, Anne T

Volltext

Ketone Bodies Mediate Antiseizure Effects von Krueger, Jena M, Nordli, Jr, Douglas R

Volltext

Remission of Childhood-onset Epilepsy von Krueger, Jena M, Nordli, Jr, Douglas R

Volltext

Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 3 Copies of SMN2 (4163) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, Macek, Thomas

Volltext

Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 2 Copies of SMN2 (4190) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, Macek, Tom

Volltext

Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with Three Copies of SMN2 (P15-5.001) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Finkel, Richard, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, McGill, Bryan, Macek, Thomas

Volltext

Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and Two Copies of SMN2 (S39.004) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Finkel, Richard, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, McGill, Bryan, Macek, Thomas

Volltext

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy von Mercuri, Eugenio, Darras, Basil T, Chiriboga, Claudia A, Day, John W, Campbell, Craig, Connolly, Anne M, Iannaccone, Susan T, Kirschner, Janbernd, Kuntz, Nancy L, Saito, Kayoko, Shieh, Perry B, Tulinius, Már, Mazzone, Elena S, Montes, Jacqueline, Bishop, Kathie M, Yang, Qingqing, Foster, Richard, Gheuens, Sarah, Bennett, C. Frank, Farwell, Wildon, Schneider, Eugene, De Vivo, Darryl C, Finkel, Richard S

Volltext

Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations von Schroth, Mary K, Deans, Jennifer, Bharucha Goebel, Diana X, Burnette, W Bryan, Darras, Basil T, Elsheikh, Bakri H, Felker, Marcia V, Klein, Andrea, Krueger, Jena, Proud, Crystal M, Veerapandiyan, Aravindhan, Graham, Robert J

Volltext

Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy in Presymptomatic Spinal Muscular Atrophy: SPR1NT Study Update (2384) von Strauss, Kevin A., Farrar, Michelle A., Swoboda, Kathryn J., Saito, Kayoko, Chiriboga, Claudia A., Finkel, Richard S., Iannaccone, Susan T., Krueger, Jena M., Kwon, Jennifer M., McMillan, Hugh J., Servais, Laurent, Mendell, Jerry R., Parsons, Julie, Scoto, Mariacristina, Shieh, Perry B., Zaidman, Craig, Schultz, Meredith, Kernbauer, Elaine, Joshi, Shivani, Ogrinc, Francis G., Kavanagh, Sarah, Feltner, Douglas E., Tauscher-Wisniewski, Sitra, McGill, Bryan E., Sproule, Douglas M., Muntoni, Francesco

Volltext

Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians von Veerapandiyan, Aravindhan, Connolly, Anne M., Mathews, Katherine D., Nelson, Stanley, McDonald, Craig, Finkel, Richard S., Vedanarayanan, Vettaikorumakankav, Tian, Cuixia, Apkon, Susan, Parsons, Julie A., Soslow, Jonathan H., Burnette, William Bryan, Batley, Kaitlin Y., Iannaccone, Susan T., Rocha, Carolina Tesi, Flanigan, Kevin M., Bharucha‐Goebel, Diana, Wright, Sarah, Monduy, Migvis, Treidler, Simona, Kumar, Ashutosh, Kuntz, Nancy L., Rao, Vamshi K., Schrader, Rachel, Bernes, Saunder M., Stefans, Vikki Ann, Krueger, Jena M., Felker, Marcia V., Hamid, Omer Abdul, Lakhotia, Arpita, Matesanz, Susan, Ghosh, Partha S., Katz, Natalie, Abdel‐Hamid, Hoda, Laverty, Chamindra G., Lee, Bo Hoon, Harper, Amy, Ramos‐Platt, Leigh, Castro, Diana, Butterfield, Russell J., Proud, Crystal M., Zaidman, Craig M., Ciafaloni, Emma

Volltext

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial von Strauss, Kevin A, Farrar, Michelle A, Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R, Servais, Laurent, McMillan, Hugh J, Finkel, Richard S, Swoboda, Kathryn J, Kwon, Jennifer M, Zaidman, Craig M, Chiriboga, Claudia A, Iannaccone, Susan T, Krueger, Jena M, Parsons, Julie A, Shieh, Perry B, Kavanagh, Sarah, Tauscher-Wisniewski, Sitra, McGill, Bryan E, Macek, Thomas A

Volltext bestellen

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial von Strauss, Kevin A, Farrar, Michelle A, Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R, Servais, Laurent, McMillan, Hugh J, Finkel, Richard S, Swoboda, Kathryn J, Kwon, Jennifer M, Zaidman, Craig M, Chiriboga, Claudia A, Iannaccone, Susan T, Krueger, Jena M, Parsons, Julie A, Shieh, Perry B, Kavanagh, Sarah, Wigderson, Melissa, Tauscher-Wisniewski, Sitra, McGill, Bryan E, Macek, Thomas A

Volltext bestellen