Screening for calcium channel modulators in CLN3 siRNA knock down SH-SY5Y neuroblastoma cells reveals a significant decrease of intracellular calcium levels by selected L-type calc...

par an Haack, Kristina, Narayan, Srinivas B., Li, Haying, Warnock, Ashley, Tan, Lu, Bennett, Michael J.
Publié 2010

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

par Berger, Kenneth I., Kanters, Steve, Jansen, Jeroen P., Stewart, Andrew, Sparks, Susan, Haack, Kristina An, Bolzani, Anna, Siliman, Gaye, Hamed, Alaa
Publié dans J Neurol (2019)

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

par Turner-Bowker, Diane M., An Haack, Kristina, Krohe, Meaghan, Yaworsky, Andrew, Vivas, Norma, Kelly, Masami, Chatterjee, Godhuli, Chaston, Emily, Mann, Erin, Reaney, Matthew
Publié dans J Patient Rep Outcomes (2020)

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

par 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
Publié dans Ther Innov Regul Sci (2020)

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

par 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
Publié dans Ther Innov Regul Sci (2020)

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

par 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
Publié dans Genet Med (2019)

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

par 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
Publié dans PLoS One (2015)