Results 271 to 280 of about 138,072 (341)
Adults With Acid Sphingomyelinase Deficiency Have Sustained Improvements in Clinical Outcomes With up to 5 Years of Olipudase Alfa Enzyme Replacement Therapy: ASCEND Trial Final Results
Journal of Inherited Metabolic Disease, Volume 49, Issue 3, May 2026.ABSTRACT
Acid sphingomyelinase deficiency (ASMD) is a rare debilitating lysosomal storage disease resulting in multisystemic disease manifestations, significant disease burden, and early mortality for some individuals. Enzyme replacement therapy (ERT) with olipudase alfa (Xenpozyme) is the first disease‐specific treatment indicated for noncentral ...Melissa P. Wasserstein, Carla E. Hollak, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto Bernardo Guelbert, Julia B. Hennermann, Takayuki Ikezoe, Robin Lachmann, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Beth L. Thurberg, Jesus Villarrubia, Nicole M. Armstrong, Shashi Bhushan, Yong Kim, Monica Kumar +20 morewiley +1 more sourceSplenectomy in β‐thalassemia patients: Practices and risks in a nationwide study
HemaSphere, Volume 10, Issue 5, May 2026.Audrey Benoit, Justine Pages, Sarah Szepetowski, Imane Agouti, Isabelle Thuret, Enora Le Roux, Catherine Badens, Valentine Brousse, NaThalY Network, Catherine Mathey, Louisa Bey, Marie‐Josée Lucchini, Valérie Li Thio Te, Reda Garidi, Loïc Garcon, Corentin Orvain, Jean‐François Brasme, Claire Reynes, Marie Belloy, Julie Durin, Oula Alhindy, Lara Sabbagh, Borhane Slama, Anthony Sanchez, Alice Moulin, Danielle Belgodere, Lise Benjemia, Pauline Simon, Eric Oziol, Nathalie Aladjidi, Yoann Huguenin, Charlotte Jubert, Edouard Forcade, Pierre Duffau, Abdallah Maakaroun, Florence Lachenal, Charline Pegon, Marie‐Helene Odievre, Charlotte Petitdidier, Sai Atmani, Florence Raqbi, Corinne Pondarre, Jenny Youn, Annie Kamdem, Dora Bachir, Pablo Bartolucci, Gonzalo De Luna, Anoosha Habibi, Elena Fois, Antoine Leblanc, Pierre‐Yves Dides, Caroline Makowski, Corinne Armari, Narcisse Elenga, Christelle Chantalat‐Auger, Françoise Driss, Ferielle Zenchri, Corinne Guitton, Mathilde Jehanne, Cecile Stoven, Axel Chaminade, Stephanie Gourdon, Yves Reguerre, Edouard Randriamalala, Véronique Paumier‐Bonjour, Anne Besancon‐Bergelin, Anne Lambilliotte, Benedicte Bruno, Fanny Gonzales, Melissa Barbati, Benjamin Carpentier, Christophe Piguet, Mohamed Touati, Arnaud Hot, Fiorenza Barraco, Giovanna Cannas, Emilie Virot, Romain Fort, Grégoire Ducoux, Hélène Desmurs Clavel, Kamila Kebaili, Nathalie Garnier, Cécile Renard, Yves Bertrand, Anne‐Laure Peugnet, Carine Halfon‐Domenech, Judith Diakiesse, Kokou‐Placide Agbo‐Kpati, Nathalie Ravet, Isabelle Thuret, Sarah Szepetowski, Estelle Jean, Benjamin De Sainte Marie, Benoit Meunier, Benoit Faucher, Matias Muszlak, Abdourahim Chamouine, Alain Garou, Caroline Masserot‐Lureau, Jean‐Marc Schneider, Veronique Dorvaux, Philippe Carassou, Honorat Andriamihaja, Muriel Lalande, Perrine Mahe, Anne Sirvent, Patricia Aguilar Martinez, Ivan Bertchansky, Jean‐Claude Eisenmann, Agathe Masseau, Marie‐Laure Couec, Fanny Rialland, Pierre‐Simon Rohrlich, Sandrine Baron‐Joly, Mohamed Conde, Antoine Dossier, Valentine Brousse, Karima Yakouben, Malika Benkerrou, Ghislaine Ithier, Florence Missud, Jean‐Hugues Dalle, Bérengène Koehl, Laurent Holvoet, Jean‐Antoine Ribeil, Laure Joseph, Marina Cavazzana, Marianne De Montalembert, Benedicte Neven, Despina Moshous, Mélissa Taylor, Slimane Allali, Bruno Varet, Gerard Socie, Alienor Xhaard, Régis Peffault De La Tour, Thierry Leblanc, Nicolas Boissel, Marie Robin, Nathalie Dhedin, Robert Girot, Sarah Mattioni, François Lionnet, Jean Donadieu, Guy Leverger, Sylvie Fasola, Jean‐Benoit Arlet, Corina Manoli, Maryse Etienne‐Julan, Emmanuelle Bernit, Justine Gellen‐Dautremer, Stéphanie Eyssette, Gilles Blondin, Claire Pluchart, Stanislas Nimubona, Jacinthe Bonneau, Sophie Bayart, Sophie Pertuisel, Benedicte Collet, Laurence Detourmignies, Julie Machin, Mathieu Wemeau, Laurence De Tourmigni, Jean‐Pierre Vannier, Bruno Filhon, Cecile Dumesnil, Agnes Lahary, Pascale Schneider, Stéphanie Ngo, Aurore Malric, Maud Schoeny, Claire Berger, Isabelle Guichard, Aurélie Desbree, Jean‐Louis Stephan, Audrey David, Catherine Paillard, Alexandra Salmon, Shanti Ame, Philippe Gestas, Marianne Besnard, Marie‐Pierre Castex, Geneviève Plat‐Wilson, Pierre Cougoul, Marlène Pasquet, Jill Serre, Marion Yvert, Jean‐Baptiste Valentin, Francis Duchene, Suzanne Mathieu, Marie Drouilly, Marie Detrait, Cécile Pochon, Aurélie Phulpin, Sylvie Nathanson +194 morewiley +1 more sourceCase Report: Co-existence of <i>BCR::PDGFRA</i> gene fusion and <i>PDGFRA</i> variants in myeloid neoplasm with persistent leukocytosis, large splenomegaly, and eosinophilia. [PDF]
Front OncolXiao Z, Lu C, Zhang P, Han N, He X, Zhang J, Feng Y, Guan M, Ouyang L, Gao Y, Li Y. +10 moreeuropepmc +1 more source
