Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y

por Jones, Simon A., Bialer, Martin, Parini, Rossella, Martin, Ken, Wang, Hui, Yang, Ke, Shaywitz, Adam J., Harmatz, Paul
Publicado 2015

Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome

por Qi, Yulan, Musson, Donald G., Schweighardt, Becky, Tompkins, Troy, Jesaitis, Lynne, Shaywitz, Adam J., Yang, Ke, O’Neill, Charles A.
Publicado 2014

Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

por Ghosh, Arunabha, Shapiro, Elsa, Rust, Stewart, Delaney, Kathleen, Parker, Samantha, Shaywitz, Adam J, Morte, Adelaida, Bubb, Gillian, Cleary, Maureen, Bo, Tien, Lavery, Christine, Bigger, Brian W, Jones, Simon A
Publicado 2017

Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double‐blind, pilot study

por Burton, Barbara K., Berger, Kenneth I., Lewis, Gregory D., Tarnopolsky, Mark, Treadwell, Marsha, Mitchell, John J., Muschol, Nicole, Jones, Simon A., Sutton, V. Reid, Pastores, Gregory M., Lau, Heather, Sparkes, Rebecca, Genter, Fred, Shaywitz, Adam J., Harmatz, Paul
Publicado 2015

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

por Davidson, Beverly L., Gao, Guangping, Berry-Kravis, Elizabeth, Bradbury, Allison M., Bönnemann, Carsten, Buxbaum, Joseph D., Corcoran, Gavin R., Gray, Steven J., Gray-Edwards, Heather, Kleiman, Robin J., Shaywitz, Adam J., Wang, Dan, Zoghbi, Huda Y., Flotte, Terence R., Tauscher-Wisniewski, Sitra, Tifft, Cynthia J., Sahin, Mustafa
Publicado 2022

A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B

por Muschol, Nicole, Koehn, Anja, von Cossel, Katharina, Okur, Ilyas, Ezgu, Fatih, Harmatz, Paul, de Castro Lopez, Maria J., Couce, Maria Luz, Lin, Shuan-Pei, Batzios, Spyros, Cleary, Maureen, Solano, Martha, Nestrasil, Igor, Kaufman, Brian, Shaywitz, Adam J., Maricich, Stephen M., Kuca, Bernice, Kovalchin, Joseph, Zanelli, Eric
Publicado 2023

Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatm...

por Grover, Anita, Crippen-Harmon, Danielle, Nave, Lacey, Vincelette, Jon, Wait, Jill C. M., Melton, Andrew C., Lawrence, Roger, Brown, Jillian R., Webster, Katherine A., Yip, Bryan K., Baridon, Brian, Vitelli, Catherine, Rigney, Sara, Christianson, Terri M., Tiger, Pascale M. N., Lo, Melanie J., Holtzinger, John, Shaywitz, Adam J., Crawford, Brett E., Fitzpatrick, Paul A., LeBowitz, Jonathan H., Bullens, Sherry, Aoyagi-Scharber, Mika, Bunting, Stuart, O’Neill, Charles A., Pinkstaff, Jason, Bagri, Anil
Publicado 2020