Using real world data to support regulatory approval of drugs in rare diseases: A review of opportunities, limitations & a case example von Gross, Andrea M.

Volltext

Selumetinib in Children with Inoperable Plexiform Neurofibromas von Gross, Andrea M, Wolters, Pamela L, Dombi, Eva, Baldwin, Andrea, Whitcomb, Patricia, Fisher, Michael J, Weiss, Brian, Kim, AeRang, Bornhorst, Miriam, Shah, Amish C, Martin, Staci, Roderick, Marie C, Pichard, Dominique C, Carbonell, Amanda, Paul, Scott M, Therrien, Janet, Kapustina, Oxana, Heisey, Kara, Clapp, D. Wade, Zhang, Chi, Peer, Cody J, Figg, William D, Smith, Malcolm, Glod, John, Blakeley, Jaishri O, Steinberg, Seth M, Venzon, David J, Doyle, L. Austin, Widemann, Brigitte C

Volltext

Longitudinal evaluation of peripheral nerve sheath tumors in neurofibromatosis type 1: growth analysis of plexiform neurofibromas and distinct nodular lesions von Akshintala, Srivandana, Baldwin, Andrea, Liewehr, David J, Goodwin, Anne, Blakeley, Jaishri O, Gross, Andrea M, Steinberg, Seth M, Dombi, Eva, Widemann, Brigitte C

Volltext

Current status of MEK inhibitors in the treatment of plexiform neurofibromas von Gross, Andrea M., Dombi, Eva, Widemann, Brigitte C.

Volltext

Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1 von Gross, Andrea M, Singh, Gurbani, Akshintala, Srivandana, Baldwin, Andrea, Dombi, Eva, Ukwuani, Somto, Goodwin, Anne, Liewehr, David J, Steinberg, Seth M, Widemann, Brigitte C

Volltext

A Monosaccharide Residue Is Sufficient to Maintain Mouse and Human IgG Subclass Activity and Directs IgG Effector Functions to Cellular Fc Receptors von Kao, Daniela, Danzer, Heike, Collin, Mattias, Groß, Andrea, Eichler, Jutta, Stambuk, Jerko, Lauc, Gordan, Lux, Anja, Nimmerjahn, Falk

Volltext

Synthetic Peptides as Protein Mimics von Groß, Andrea, Hashimoto, Chie, Sticht, Heinrich, Eichler, Jutta

Volltext

Neurofibromatosis Clinical Trials—REiNS Collaboration 2020 Recommendations: Looking Back and Moving Ahead von Gross, Andrea M., Plotkin, Scott R., Widemann, Brigitte C.

Volltext

A molecular basis for neurofibroma-associated skeletal manifestations in NF1 von Ma, Yun, Gross, Andrea M., Dombi, Eva, Pemov, Alexander, Choi, Kwangmin, Chaney, Katherine, Rhodes, Steven D., Angus, Steven P., Sciaky, Noah, Clapp, D. Wade, Ratner, Nancy, Widemann, Brigitte C., Rios, Jonathan J., Elefteriou, Florent

Volltext

Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas von Gross, Andrea M, Dombi, Eva, Wolters, Pamela L, Baldwin, Andrea, Dufek, Anne, Herrera, Kailey, Martin, Staci, Derdak, Joanne, Heisey, Kara S, Whitcomb, Patricia M, Steinberg, Seth M, Venzon, David J, Fisher, Michael J, Kim, AeRang, Bornhorst, Miriam, Weiss, Brian D, Blakeley, Jaishri O, Smith, Malcolm A, Widemann, Brigitte C

Volltext

Novel tube-type LTCC transducers with buried heaters and inner interdigitated electrodes as a platform for gas sensing at various high temperatures von Brandenburg, Annica, Kita, Jaroslaw, Groß, Andrea, Moos, Ralf

Volltext

Anthropometric measurements of children with neurofibromatosis type I: impact of plexiform neurofibroma volume and treatment von Lemberg, Kathryn M, Gross, Andrea M, Sproule, Lauren M, Liewehr, David J, Dombi, Eva, Baldwin, Andrea, Steinberg, Seth M, Bornhorst, Miriam, Lodish, Maya, Blakeley, Jaishri O, Widemann, Brigitte C

Volltext

Selumetinib in children with neurofibromatosis type 1 and asymptomatic inoperable plexiform neurofibroma at risk for developing tumor-related morbidity von Gross, Andrea M, Glassberg, Brittany, Wolters, Pamela L, Dombi, Eva, Baldwin, Andrea, Fisher, Michael J, Kim, AeRang, Bornhorst, Miriam, Weiss, Brian D, Blakeley, Jaishri O, Whitcomb, Patricia, Paul, Scott M, Steinberg, Seth M, Venzon, David J, Martin, Staci, Carbonell, Amanda, Heisey, Kara, Therrien, Janet, Kapustina, Oxana, Dufek, Anne, Derdak, Joanne, Smith, Malcolm A, Widemann, Brigitte C

Volltext

MEK inhibitors for neurofibromatosis type 1 manifestations: Clinical evidence and consensus von de Blank, Peter M K, Gross, Andrea M, Akshintala, Srivandana, Blakeley, Jaishri O, Bollag, Gideon, Cannon, Ashley, Dombi, Eva, Fangusaro, Jason, Gelb, Bruce D, Hargrave, Darren, Kim, AeRang, Klesse, Laura J, Loh, Mignon, Martin, Staci, Moertel, Christopher, Packer, Roger, Payne, Jonathan M, Rauen, Katherine A, Rios, Jonathan J, Robison, Nathan, Schorry, Elizabeth K, Shannon, Kevin, Stevenson, David A, Stieglitz, Elliot, Ullrich, Nicole J, Walsh, Karin S, Weiss, Brian D, Wolters, Pamela L, Yohay, Kaleb, Yohe, Marielle E, Widemann, Brigitte C, Fisher, Michael J

Volltext

Audiometric and Otologic Findings in Children and Young Adults with Neurofibromatosis Type 1 and Plexiform Neurofibromas von Idowu, Victoria, Christensen, Julie, Gross, Andrea M, Dombi, Eva, Miles, Jennifer R, King, Kelly, Chisholm, Jennifer, Zalewski, Christopher, Baldwin, Andrea, Whitcomb, Patricia, Burgess, Crystal, Widemann, Brigitte C, Brewer, Carmen C, Kim, Hung Jeffrey

Volltext

Advancing neurofibromatosis and schwannomatosis clinical trial design: Consensus recommendations from the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) Inter... von Merker, Vanessa L, Gross, Andrea M, Widemann, Brigitte C, Plotkin, Scott R

Volltext

Croatian Action on Salt and Health (CRASH): On the Road to Success-Less Salt, More Health von Jelaković, Bojan, Marinović Glavić, Mihaela, Batinić Sermek, Marija, Bilajac, Lovorka, Bubaš, Marija, Buzjak Služek, Vlatka, Capak, Krunoslav, Drenjančević, Ines, Gross Bošković, Andrea, Jelaković, Ana, Jukić, Tomislav, Kolarić Kravar, Sanja, Kralj, Verica, Pećin, Ivan, Pollak, Lea, Skoko-Poljak, Dunja, Stražanac, Danijela, Stupin, Ana, Vasiljev, Vanja, Vidranski, Valentina, Reiner, Željko

Volltext

Element content in ten Croatian honey types from different geographical regions during three seasons von Bilandžić, Nina, Sedak, Marija, Đokić, Maja, Bošković, Andrea Gross, Florijančić, Tihomir, Bošković, Ivica, Kovačić, Marin, Puškadija, Zlatko, Hruškar, Mirjana

Volltext

The Landscape of US and Global Rare Tumor Research Programs: A Systematic Review von Vivelo, Christina, Reilly, Karlyne M, Widemann, Brigitte C, Wedekind, Mary Frances, Painter, Corrie, O’Neill, Allison F, Mueller, Sabine, Elemento, Olivier, Gross, Andrea M, Sandler, Abby B

Volltext

Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial von Gross, Andrea M., O’Sullivan Coyne, Geraldine, Dombi, Eva, Tibery, Cecilia, Herrick, William G., Martin, Staci, Angus, Steven P., Shern, Jack F., Rhodes, Steven D., Foster, Jared C., Rubinstein, Larry V., Baldwin, Andrea, Davis, Christopher, Dixon, Shelley A. H., Fagan, Margaret, Ong, Mary Jane, Wolters, Pamela L., Tamula, Mary Anne, Reid, Olivia, Sankaran, Hari, Fang, Fang, Govindharajulu, Jeevan Prasaad, Browne, Alice T., Kaplan, Rosandra N., Heisey, Kara, On, Thomas J., Xuei, Xiaoling, Zhang, Xiyuan, Johnson, Barry C., Parchment, Ralph E., Clapp, D. Wade, Srivastava, Apurva K., Doroshow, James H., Chen, Alice P., Widemann, Brigitte C.

Volltext