Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective

por Hamed, Alaa, An Haack, Kristina, Gwaltney, Chad, Baranowski, Eileen, Stewart, Andrew, Krupnick, Robert, Tyler, Margaret, Sparks, Susan, Paty, Jean
Publicado 2021

Forced vital capacity and cross-domain late-onset Pompe disease outcomes: an individual patient-level data meta-analysis

por Berger, Kenneth I., Kanters, Steve, Jansen, Jeroen P., Stewart, Andrew, Sparks, Susan, Haack, Kristina An, Bolzani, Anna, Siliman, Gaye, Hamed, Alaa
Publicado 2019

Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease

por Tiraboschi, Gilles, Marchionni, David, Tuffal, Gilles, Fabre, David, Martinez, Jean-Marie, Haack, Kristina An, Miossec, Patrick, Kittner, Barbara, Daba, Nadia, Hurbin, Fabrice
Publicado 2023

Development and content validation of the Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQ): patient-reported and caregiver-reported outcome measures

por Turner-Bowker, Diane M., An Haack, Kristina, Krohe, Meaghan, Yaworsky, Andrew, Vivas, Norma, Kelly, Masami, Chatterjee, Godhuli, Chaston, Emily, Mann, Erin, Reaney, Matthew
Publicado 2020

How is the Pharmaceutical Industry Structured to Optimize Pediatric Drug Development? Existing Pediatric Structure Models and Proposed Recommendations for Structural Enhancement

por Severin, Thomas, Corriol-Rohou, Solange, Bucci-Rechtweg, Christina, an Haack, Kristina, Fuerst-Recktenwald, Sabine, Lepola, Pirkko, Norjavaara, Ensio, Dehlinger-Kremer, Martine, Haertter, Sebastian, Cheung, S. Y. Amy
Publicado 2020

Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study

por Dimachkie, Mazen M., Kishnani, Priya S., Ivanescu, Cristina, Flore, Giulio, Gwaltney, Chad, van der Beek, Nadine A.M.E., Hamed, Alaa, An Haack, Kristina, Pollissard, Laurence, Baranowski, Eileen, Sparks, Susan E., DasMahapatra, Pronabesh
Publicado 2023

Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort

por Duong, Tina, Kishnani, Priya S., An Haack, Kristina, Foster, Meredith C., Gibson, James B., Wilson, Catherine, Hahn, Si Houn, Hillman, Richard, Kronn, David, Leslie, Nancy D., Peña, Loren D.M., Sparks, Susan E., Stockton, David W., Tanpaiboon, Pranoot, Day, John W.
Publicado 2022

Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease

por Kishnani, Priya S., Gibson, James B., Gambello, Michael J., Hillman, Richard, Stockton, David W., Kronn, David, Leslie, Nancy D., Pena, Loren D. M., Tanpaiboon, Pranoot, Day, John W., Wang, Raymond Y., Goldstein, Jennifer L., An Haack, Kristina, Sparks, Susan E., Zhao, Yang, Hahn, Si Houn
Publicado 2019

Characterization of Early Disease Status in Treatment-Naive Male Paediatric Patients with Fabry Disease Enrolled in a Randomized Clinical Trial

por Wijburg, Frits A., Bénichou, Bernard, Bichet, Daniel G., Clarke, Lorne A., Dostalova, Gabriela, Fainboim, Alejandro, Fellgiebel, Andreas, Forcelini, Cassiano, An Haack, Kristina, Hopkin, Robert J., Mauer, Michael, Najafian, Behzad, Scott, C. Ronald, Shankar, Suma P., Thurberg, Beth L., Tøndel, Camilla, Tylki-Szymańska, Anna, Ramaswami, Uma
Publicado 2015

Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease

por Dimachkie, Mazen M., Barohn, Richard J., Byrne, Barry, Goker-Alpan, Ozlem, Kishnani, Priya S., Ladha, Shafeeq, Laforêt, Pascal, Mengel, Karl Eugen, Peña, Loren D.M., Sacconi, Sabrina, Straub, Volker, Trivedi, Jaya, Van Damme, Philip, van der Ploeg, Ans T., Vissing, John, Young, Peter, Haack, Kristina An, Foster, Meredith, Gilbert, Jane M., Miossec, Patrick, Vitse, Olivier, Zhou, Tianyue, Schoser, Benedikt
Publicado 2022

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial

por Kishnani, Priya S., Diaz-Manera, Jordi, Toscano, Antonio, Clemens, Paula R., Ladha, Shafeeq, Berger, Kenneth I., Kushlaf, Hani, Straub, Volker, Carvalho, Gerson, Mozaffar, Tahseen, Roberts, Mark, Attarian, Shahram, Chien, Yin-Hsiu, Choi, Young-Chul, Day, John W., Erdem-Ozdamar, Sevim, Illarioshkin, Sergey, Goker-Alpan, Ozlem, Kostera-Pruszczyk, Anna, van der Ploeg, Ans T., An Haack, Kristina, Huynh-Ba, Olivier, Tammireddy, Swathi, Thibault, Nathan, Zhou, Tianyue, Dimachkie, Mazen M., Schoser, Benedikt
Publicado 2023