Mutations in KIT and RAS are frequent events in pediatric core-binding factor acute myeloid leukemia von GOEMANS, B. F, ZWAAN, Ch. M, KASPERS, G. J. L, HEINRICH, M. C, MILLER, M, ZIMMERMANN, M, HARLOW, A, MESHINCHI, S, LOONEN, A. H, HIHLEN, K, REINHARDT, D, CREUTZIG, U

Volltext

Stability and prognostic influence of FLT3 mutations in paired initial and relapsed AML samples von CLOOS, J, GOEMANS, B. F, CREUTZIG, U, SCHUURHUIS, G. J, ZWAAN, Ch. M, KASPERS, G. J. L, HESS, C. J, VAN OOSTVEEN, J. W, WAISFISZ, Q, CORTHALS, S, DE LANGE, D, BOECKX, N, HÄHLEN, K, REINHARDT, D

Volltext

Increased risk of leukaemia in children with Down syndrome: a somatic evolutionary view von Hasaart, K. A. L., Bertrums, E. J. M., Manders, F., Goemans, B. F., van Boxtel, R.

Volltext

Management of cerebral azole-resistant Aspergillus fumigatus infection: A role for intraventricular liposomal-amphotericin B von Schauwvlieghe, A.F.A.D., Bredius, R.G.M., Verdijk, R.M., Smiers, F.J.W., van der Beek, M.T., Goemans, B.F., Zwaan, C.M., Brüggemann, R.J., Rijnders, B.J.A.

Volltext

Differences in the prevalence of PTPN11 mutations in FAB M5 paediatric acute myeloid leukaemia von Goemans, B. F., Zwaan, Ch. M., Martinelli, S., Harrell, P., Lange, D., Carta, C., Reinhardt, D., Hählen, K., Creutzig, U., Tartaglia, M., Heinrich, M. C., Kaspers, G. J. L.

Volltext

Large interindividual differences in cellular sensitivity to calicheamicin may influence gemtuzumab ozogamicin response in acute myeloid leukemia von Goemans, B F, Zwaan, C M, Vijverberg, S J H, Loonen, A H, Creutzig, U, Hählen, K, Reinhardt, D, Gibson, B E S, Cloos, J, Kaspers, G J L

Volltext

Clinical outcomes of second relapsed and refractory first relapsed paediatric AML: A retrospective study within the NOPHO‐DB SHIP consortium von White, Tara, Kaspers, Gertjan, Abrahamsson, Jonas, Arad‐Cohen, Nira, Cianci, Daniela, Fernandez, Jose, Ha, Shau‐Yin, Hasle, Henrik, De Moerloose, Barbara, Zwaan, C. Michel, Goemans, Bianca F.

Volltext

In vitro profiling of the sensitivity of pediatric leukemia cells to tipifarnib: identification of T-cell ALL and FAB M5 AML as the most sensitive subsets von Goemans, Bianca F., Zwaan, Christian M., Harlow, Amy, Loonen, Anne H., Gibson, Brenda E.S., Hählen, Karel, Reinhardt, Dirk, Creutzig, Ursula, Heinrich, Michael C., Kaspers, Gertjan J.L.

Volltext

Outcome for children with relapsed acute myeloid leukemia in the Netherlands following initial treatment between 1980 and 1998: survival after chemotherapy only? von Goemans, Bianca F, Tamminga, Rienk Y J, Corbijn, Carin M, Hählen, Karel, Kaspers, Gertjan J L

Volltext

Management of cerebral azole-resistant Aspergillus fumigatus infection : a role for intraventricular liposomal-amphotericin B von Schauwvlieghe, Alexander, Bredius, R. G. M, Verdijk, R. M, Smiers, F. J. W, van der Beek, M. T, Goemans, B. F, Zwaan, C. M, Bruggemann, R. J, Rijnders, B. J. A

Volltext

A comparison of the in vitro cytotoxicity of daunorubicin and liposomal daunorubicin in pediatric acute leukemia von Goemans, BF, Zwaan, CM, Reinhardt, D, Gibson, BE, Hahlen, K, Kaspers, GJ

Volltext

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy von Goemans, Nathalie M, Tulinius, Mar, van den Akker, Johanna T, Burm, Brigitte E, Ekhart, Peter F, Heuvelmans, Niki, Holling, Tjadine, Janson, Anneke A, Platenburg, Gerard J, Sipkens, Jessica A, Sitsen, J.M. Ad, Aartsma-Rus, Annemieke, van Ommen, Gert-Jan B, Buyse, Gunnar, Darin, Niklas, Verschuuren, Jan J, Campion, Giles V, de Kimpe, Sjef J, van Deutekom, Judith C

Volltext

Local Dystrophin Restoration with Antisense Oligonucleotide PRO051 von van Deutekom, Judith C, Janson, Anneke A, Ginjaar, Ieke B, Frankhuizen, Wendy S, Aartsma-Rus, Annemieke, Bremmer-Bout, Mattie, den Dunnen, Johan T, Koop, Klaas, van der Kooi, Anneke J, Goemans, Nathalie M, de Kimpe, Sjef J, Ekhart, Peter F, Venneker, Edna H, Platenburg, Gerard J, Verschuuren, Jan J, van Ommen, Gert-Jan B

Volltext

Measurable Residual Disease and Fusion Partner Independently Predict Survival and Relapse Risk in Childhood KMT2A -Rearranged Acute Myeloid Leukemia: A Study by the International B... von van Weelderen, Romy E, Klein, Kim, Harrison, Christine J, Jiang, Yilin, Abrahamsson, Jonas, Arad-Cohen, Nira, Bart-Delabesse, Emmanuelle, Buldini, Barbara, De Moerloose, Barbara, Dworzak, Michael N, Elitzur, Sarah, Fernández Navarro, José M, Gerbing, Robert B, Goemans, Bianca F, de Groot-Kruseman, Hester A, Guest, Erin, Ha, Shau-Yin, Hasle, Henrik, Kelaidi, Charikleia, Lapillonne, Hélène, Leverger, Guy, Locatelli, Franco, Masetti, Riccardo, Miyamura, Takako, Norén-Nyström, Ulrika, Polychronopoulou, Sophia, Rasche, Mareike, Rubnitz, Jeffrey E, Stary, Jan, Tierens, Anne, Tomizawa, Daisuke, Zwaan, C Michel, Kaspers, Gertjan J L

Volltext

Implementation of paediatric precision oncology into clinical practice: The Individualized Therapies for Children with cancer program ‘iTHER’ von Langenberg, Karin P.S., Meister, Michael T., Bakhuizen, Jette J., Boer, Judith M., van Eijkelenburg, Natasha K.A., Hulleman, Esther, Ilan, Uri, Looze, Eleonora J., Dierselhuis, Miranda P., van der Lugt, Jasper, Breunis, Willemijn, Schild, Linda G., Ober, Kimberley, van Hooff, Sander R., Scheijde-Vermeulen, Marijn A., Hiemcke-Jiwa, Laura S., Flucke, Uta E., Kranendonk, Mariette E.G., Wesseling, Pieter, Sonneveld, Edwin, Punt, Simone, Boltjes, Arjan, van Dijk, Freerk, Verwiel, Eugene T.P., Volckmann, Richard, Hehir-Kwa, Jayne Y., Kester, Lennart A., Koudijs, Marco M.J., Waanders, Esme, Holstege, Frank C.P., Vormoor, H. Josef, Hoving, Eelco W., van Noesel, Max M., Pieters, Rob, Kool, Marcel, Stumpf, Miriam, Blattner-Johnson, Mirjam, Balasubramanian, Gnana P., Van Tilburg, Cornelis M., Jones, Barbara C., Jones, David T.W., Witt, Olaf, Pfister, Stefan M., Jongmans, Marjolijn C.J., Kuiper, Roland P., de Krijger, Ronald R., Wijnen, Marc H.W., den Boer, Monique L., Zwaan, C. Michel, Kemmeren, Patrick, Koster, Jan, Tops, Bastiaan B.J., Goemans, Bianca F., Molenaar, Jan J.

Volltext

Characteristics and treatment of acute myeloid neoplasms with cutaneous involvement in infants up to 6 months of age: A retrospective study von Renaud, Juliette, Goemans, Bianca F., Locatelli, Franco, Pigazzi, Martina, Redmond, Shelagh, Kuehni, Claudia E., Destaillats, Alice, Alonzo, Todd A., Gerbing, Robert B., Gamis, Alan, Aplenc, Richard, Renella, Raffaele, Cooper, Todd, Ceppi, Francesco

Volltext

Ataluren treatment of patients with nonsense mutation dystrophinopathy von Bushby, Katharine, Finkel, Richard, Wong, Brenda, Barohn, Richard, Campbell, Craig, Comi, Giacomo P., Connolly, Anne M., Day, John W., Flanigan, Kevin M., Goemans, Nathalie, Jones, Kristi J., Mercuri, Eugenio, Quinlivan, Ros, Renfroe, James B., Russman, Barry, Ryan, Monique M., Tulinius, Mar, Voit, Thomas, Moore, Steven A., Lee Sweeney, H., Abresch, Richard T., Coleman, Kim L., Eagle, Michelle, Florence, Julaine, Gappmaier, Eduard, Glanzman, Allan M., Henricson, Erik, Barth, Jay, Elfring, Gary L., Reha, Allen, Spiegel, Robert J., O'donnell, Michael W., Peltz, Stuart W., Mcdonald, Craig M.

Volltext

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management von Bushby, Katharine, MD, Finkel, Richard, MD, Birnkrant, David J, MD, Case, Laura E, DPT, Clemens, Paula R, MD, Cripe, Linda, MD, Kaul, Ajay, MD, Kinnett, Kathi, MSN, McDonald, Craig, MD, Pandya, Shree, PT, Poysky, James, PhD, Shapiro, Frederic, MD, Tomezsko, Jean, PhD, Constantin, Carolyn, PhD

Volltext

Mutations of the Selenoprotein N Gene, Which Is Implicated in Rigid Spine Muscular Dystrophy, Cause the Classical Phenotype of Multiminicore Disease: Reassessing the Nosology of Ea... von Ferreiro, Ana, Quijano-Roy, Susana, Pichereau, Claire, Moghadaszadeh, Behzad, Goemans, Nathalie, Bönnemann, Carsten, Jungbluth, Heinz, Straub, Volker, Villanova, Marcello, Leroy, Jean-Paul, Romero, Norma B., Martin, Jean-Jacques, Muntoni, Francesco, Voit, Thomas, Estournet, Brigitte, Richard, Pascale, Fardeau, Michel, Guicheney, Pascale

Volltext

P.29 Accelerating clinical development of new therapeutics with patient data: evidence from the collaborative Trajectory Analysis Program (cTAP) in DMD von McDonald, C., Ward, S., Signorovitch, J., Muntoni, F., Goemans, N., Wong, B., Vandenborne, K., Manzur, A., Sajeev, G., Mercuri, E.

Volltext