Results 201 to 210 of about 5,785,217 (307)
ABSTRACT The survival of infants and children with complex medical conditions continues to increase, underscoring the need to quantify technology dependence more effectively. Existing measures emphasize mortality risk and fail to capture the burden of ongoing technological support.
Ashlee J. Vance +5 more
wiley +1 more source
Late effects after allogeneic hematopoietic stem cell transplantation in patients with primary immunodeficiency. [PDF]
Grubliauskaitė G +2 more
europepmc +1 more source
: Background. Human metapneumovirus (hMPV) epidemiology, clinical characteristics and risk factors for poor outcome after allogeneic stem cell transplantation (allo-HCT) remain a poorly investigated area. Methods.
Wachowiak, Jacek +43 more
core
Hemophilia A: An Ideal Disease for Prenatal Therapy
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada +2 more
wiley +1 more source
Diagnosis and Management of Prenatal Hereditary Pyropoikilocytosis
ABSTRACT Hereditary pyropoikilocytosis (HPP) is a severe hemolytic anemia caused by variants in SPTA1, SPTB, and EPB41. These weaken horizontal interactions in the erythrocyte cytoskeleton, causing membrane fragmentation and splenic sequestration. It will readily cause fetal anemia and often hydrops fetalis. Prenatal diagnosis requires first ruling out
Connor Hartzell +6 more
wiley +1 more source
Attitudes Toward Prenatal Interventions in the Fanconi Anemia Community
ABSTRACT Objective In‐utero cell and gene therapies may offer prenatal treatment options for inherited diseases. Preclinical data suggests in‐utero (IU) hematopoietic stem cell transplantation (HSCT) could prevent Fanconi anemia (FA) related bone marrow failure without genotoxic conditioning or immune suppression.
Tony Lum +4 more
wiley +1 more source
The feasibility of ovarian tissue cryopreservation in selected females after hematopoietic stem cell transplantation. [PDF]
Cheng J +15 more
europepmc +1 more source
ABSTRACT Sickle cell disease (SCD) affects millions worldwide but has limited treatment options, most of which carry significant side effects. At present, the only curative treatment for SCD is allogeneic or gene‐modified autologous hematopoietic stem cell (HSC) transplantation (Tx).
Oluwaseun O. Babatunde +4 more
wiley +1 more source
Microbiota injury during conditioning for allogeneic hematopoietic stem cell transplantation. [PDF]
Lipan LE +7 more
europepmc +1 more source

