Results 11 to 20 of about 415,260 (411)

The clinical spectrum of SMA‐PME and in vitro normalization of its cellular ceramide profile

Annals of Clinical and Translational Neurology, Volume 9, Issue 12, Page 1941-1952, December 2022., 2022
Abstract Objective The objectives of this study were to define the clinical and biochemical spectrum of spinal muscular atrophy with progressive myoclonic epilepsy (SMA‐PME) and to determine if aberrant cellular ceramide accumulation could be normalized by enzyme replacement.
Michelle M. Lee, Graeme S. V. McDowell, Darryl C. De Vivo, Daniel Friedman, Samuel F. Berkovic, Maria Spanou, Argirios Dinopoulos, Katheryn Grand, Pedro A. Sanchez‐Lara, Michelle Allen‐Sharpley, Jodi Warman‐Chardon, Alexander Solyom, Thierry Levade, Edward H. Schuchman, Steffany A. L. Bennett, David A. Dyment, Toni S. Pearson   +16 more
wiley   +1 more source

Recognizing early MRI signs (or their absence) is crucial in diagnosing metachromatic leukodystrophy

Annals of Clinical and Translational Neurology, Volume 9, Issue 12, Page 1999-2009, December 2022., 2022
Abstract Objectives Metachromatic leukodystrophy (MLD) has characteristic white matter (WM) changes on brain MRI, which often trigger biochemical and genetic confirmation of the diagnosis. In early or pre‐symptomatic disease stages, these typical MRI changes might be absent, hampering early diagnosis.
Daphne H. Schoenmakers, Shanice Beerepoot, Ingeborg Krägeloh‐Mann, Saskia Elgün, Benjamin Bender, Marjo S. van der Knaap, Nicole I. Wolf, Samuel Groeschel   +7 more
wiley   +1 more source

Early relapse prediction after allogeneic hematopoietic stem cell transplantation for acute lymphoblastic leukemia (ALL) using lineage‐specific chimerism analysis

eJHaem, 2022
Relapse is a major cause of treatment failure after hematopoietic stem cell transplantation (HSCT) for acute leukemia. Here, we report a monocentric retrospective study of all HSCTs for B cell acute lymphoblastic leukemia (ALL) performed during the years
Hannes Lindahl, Davide Valentini, Sofie Vonlanthen, Mikael Sundin, Andreas T. Björklund, Stephan Mielke, Dan Hauzenberger   +6 more
doaj   +1 more source

Rabbit ATG/ATLG in preventing graft-versus-host disease after allogeneic stem cell transplantation: consensus-based recommendations by an international expert panel

Bone Marrow Transplantation, 2020
This collaborative initiative aimed to provide recommendations on the use of polyclonal antithymocyte globulin (ATG) or anti-T lymphocyte globulin (ATLG) for the prevention of graft-versus-host disease (GvHD) after allogeneic hematopoietic stem cell ...
F. Bonifazi, M. Rubio, A. Bacigalupo, J. Boelens, J. Finke, H. Greinix, M. Mohty, A. Nagler, J. Passweg, A. Rambaldi, G. Socié, C. Solano, I. Walker, G. Barosi, N. Kröger   +14 more
semanticscholar   +1 more source

The Importance of Secretor-Status in Norovirus Infection Following Allogeneic Hematopoietic Stem Cell Transplantation

Viruses, 2022
Background. Human secretor-status is a strong susceptibility factor for norovirus infection in immunocompetent people. The predominant norovirus genotype GII.4 almost exclusively infects secretors and is also associated with more severe symptoms. However,
Lisa Swartling, Elda Sparrelid, Per Ljungman, Ksenia Boriskina, Davide Valentini, Lennart Svensson, Johan Nordgren   +6 more
doaj   +1 more source

Low-intensity pulsed ultrasound promotes mesenchymal stem cell transplantation-based articular cartilage regeneration via inhibiting the TNF signaling pathway [PDF]

Stem Cell Res Ther 14, 93 (2023), 2021
Background: Mesenchymal stem cell (MSC) transplantation therapy is highly investigated for the regenerative repair of cartilage defects. Low-intensity pulsed ultrasound (LIPUS) has the potential to promote chondrogenic differentiation of MSCs. However, its underlying mechanism remains unclear.
arxiv   +1 more source

Allogeneic Stem Cell Transplantation in Multiple Myeloma [PDF]

Cancers, 2021
The development of new inhibitory and immunological agents and combination therapies significantly improved response rates and survival of patients diagnosed with multiple myeloma (MM) in the last decade, but the disease is still considered to be incurable by current standards and the prognosis is dismal especially in high-risk groups and in relapsed ...
Christine Greil, Monika Engelhardt, Jürgen Finke, Ralph Wäsch   +3 more
openaire   +3 more sources

Allogeneic Stem Cell Transplantation in Myelofibrosis [PDF]

Biology of Blood and Marrow Transplantation, 2017
Myeloproliferative neoplasm (MPN) is a category in the World Health Organization classification of myeloid tumors. BCR-ABL1-negative MPN is a subcategory that includes primary myelofibrosis (MF), post-essential thrombocythemia MF, and post-polycythemia vera MF. These disorders are characterized by stem cell-derived clonal myeloproliferation. Clinically,
Jeanne Palmer, Tania Jain, Ruben A. Mesa
openaire   +3 more sources

Allogeneic hematopoietic stem-cell transplantation for myelofibrosis [PDF]

Therapeutic Advances in Hematology, 2020
Myelofibrosis is one of the Philadelphia chromosome (Ph)-negative myeloproliferative neoplasms with heterogeneous clinical course. Though many treatment options, including Janus kinase (JAK) inhibitors, have provided clinical benefits and improved survival, allogeneic hematopoietic stem-cell transplantation (AHSCT) remains the only potentially ...
Lining Zhang, Fan Yang, Sizhou Feng
openaire   +4 more sources

Donor lymphocyte count and thymic activity predict lymphocyte recovery and outcomes after matched-sibling hematopoietic stem cell transplant

Haematologica, 2013
Delayed immune recovery is a characteristic feature of allogeneic hematopoietic stem cell transplantation in adult recipients. Although recipient thymic T-cell neogenesis contributes to T-cell regeneration after transplantation, thymic recovery in the ...
Zachariah McIver, Jan Joseph Melenhorst, Colin Wu, Andrew Grim, Sawa Ito, Irene Cho, Nancy Hensel, Minoo Battiwalla, Austin John Barrett   +8 more
doaj   +1 more source