Results 31 to 40 of about 3,137,982 (258)

Social Functioning Within the First Years After Pediatric Brain Tumor Diagnosis and the Relationship With Family Psychosocial Risk

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Survivors of pediatric brain tumors (PBTs) can experience long‐term social difficulties, impacting quality of life. Beyond medical and environmental factors, family psychosocial risk may play a role in social outcomes by shaping the caregiving environment and may provide intervention options.
Renske H. Houben, Marisa H. Huisman, Martha A. Grootenhuis, Femke K. Aarsen, Marita Partanen   +4 more
wiley   +1 more source

Hematopoietic Stem Cell Transplantation in CSF1R-Related Leukoencephalopathy: Retrospective Study on Predictors of Outcomes

Pharmaceutics, 2022
Mutations in the CSF1R gene are the most common cause of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a neurodegenerative disease with rapid progression and ominous prognosis.
Jarosław Dulski, Michael G. Heckman, Launia J. White, Kamila Żur-Wyrozumska, Troy C. Lund, Zbigniew K. Wszolek   +5 more
doaj   +1 more source

Pediatric Idiopathic Multicentric Castleman Disease Is Often Severe But Responsive to Siltuximab

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Idiopathic multicentric Castleman disease (iMCD) is a potentially fatal immunologic disorder marked by widespread lymphadenopathy and inflammation. Siltuximab, an interleukin‐6 (IL‐6) inhibitor, is the only FDA‐approved treatment for adult patients with iMCD.
Bridget Austin, Sheila K. Pierson, Kelley C. Dacus, Mateo Sarmiento Bustamante, Saishravan Shyamsundar, Larissa Borys, Kayla McHugh, Abiola Irvine, Yoojin Jo, Adam Bagg, Mary Jo Lechowicz, Daisy Alapat, Amy Chadburn, Megan S. Lim, Gordan Srkalovic, Frits van Rhee, David C. Fajgenbaum, Joshua D. Brandstadter   +17 more
wiley   +1 more source

Lower limb involvement in adult-onset primary dystonia: frequency and clinical features

, 2010
Despite the growing number of reports describing adult-onset primary lower limb dystonia (LLD) this entity has never been systematically evaluated in the general population of patients with primary adult-onset ...
Liuzzi, D., GIRLANDA P, Fabbrini G., LIGUORI, ROCCO, Girlanda, P., SABETTA A, Guidubaldi, Arianna, MORGANTE F, A. R. Bentivoglio, G. Fabbrini, Fabbrini, G., ABBRUZZESE G, FABBRINI G, Sabetta, A., FABBRINI, Giovanni, Abbruzzese G., Esposito M., A. Guidubaldi, Guidubaldi, A., A. Sabetta, A. Berardelli, Abbruzzese, G., Marinelli, L., MACEROLLO A, G. Defazio, DEFAZIO, Giovanni, SANTORO, LUCIO, Marinelli L., Girlanda P., SANTORO L, D. Liuzzi, Guidubaldi A., LIGUORI R, Defazio G., M. Esposito, Liuzzi D., Bentivoglio, Anna Rita, D. Martino, GUIDUBALDI A, A. Macerollo, ESPOSITO, MARCELLO, L. Marinelli, L. Santoro, Bentivoglio A. R., ESPOSITO, M, R. Liguori, Esposito M, Berardelli, A., MARTINO D, BERARDELLI A, Defazio, G., Sabetta A., Morgante, F., Martino D., LIUZZI D, Bentivoglio, A. R., Martino, D., Macerollo, A., BENTIVOGLIO AR, Liguori, R., Morgante F., P. Girlanda, ABBRUZZESE, GIOVANNI, G. Abbruzzese, MARINELLI L, F. Morgante, Macerollo A., Santoro, L., MARINELLI, LUCIO, BERARDELLI, Alfredo, Berardelli A., DEFAZIO G, Santoro L.   +72 more
core   +1 more source

Adult-onset pompe’s disease presenting with insidious hypercapnic respiratory failure [PDF]

, 2016
Orthopnoea is commonly attributed to heart failure but can be caused by diaphragm weakness, which, when severe, is often associated with hypercapnic respiratory failure.
Tsang, B, Tay, G, Masel, P, Henderson, R, O’Callaghan, C   +4 more
core   +1 more source

Chemotherapy for Spinal Cord Intramedullary Pediatric Low‐Grade Glioma—A Systematic Literature Review

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Pediatric intramedullary low‐grade gliomas (PIMLGGs) are rare neoplasms that present unique clinical and management challenges. Although surgery remains the primary tool for tissue diagnosis and decompression, complete gross‐total resection is frequently unfeasible due to the infiltrative nature of these lesions.
Olga M. Sergeenko, Segev Gabay, Jonathan Roth, Amit Ringel, Rina Dvir, Shlomi Constantini   +5 more
wiley   +1 more source

Adult-onset bulbar ptosis in Joubert syndrome [PDF]

, 2012
Benjamin Burt, Johanan Levine, Kim LeTexas Tech University, Department of Ophthalmology, Paul L Foster School of Medicine, El Paso, TexasAbstract: In this case report, we describe a case of adult-onset bulbar ptosis in a patient with Joubert syndrome ...
Le K, Burt B, Levine J
core  

Classical Complement Pathway Inhibition in a Pediatric Patient With Refractory Immune Thrombocytopenia

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Pediatric refractory immune thrombocytopenia (rITP) is generally characterized by persistent thrombocytopenia and failure to achieve a sustained response to multiple standard therapies. We report a 4‐year‐old female with congenital tufting enteropathy, post‐intestinal transplant patient on immunosuppressants, who developed severe rITP ...
Sarah Hsieh, Susmita Sarangi, Caileigh Pudela, Catherine M. Broome, Tara K. Suntum   +4 more
wiley   +1 more source

Safety of Daprodustat for the Treatment of Chronic Kidney Disease Anemia: Final Analysis of a Multicenter Postmarketing Surveillance Study in Japan

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Introduction This final analysis of a multicenter, prospective postmarketing surveillance study evaluated the safety of daprodustat in patients with chronic kidney disease anemia in routine clinical practice in Japan. Methods Patients who initiated daprodustat between September 2020 and July 2022 were registered.
Tadao Akizawa, Kyomi Kanaya, Kiyomi Aizawa, Masanori Onoki, Hirofumi Ozeki, Kazuko Suzuki, Ai Hayashi, Tomohiko Asamura   +7 more
wiley   +1 more source

Effect of Delayed Diagnosis of Phenylketonuria With Imaging Findings of Bilateral Diffuse Symmetric White Matter Lesions: A Case Report and Literature Review

Frontiers in Neurology, 2019
Phenylketonuria is a hereditary metabolic disorder due to the deficiency of tetrahydrobiopterin or phenylalanine hydroxylase. Delayed diagnoses of it manifest a progressive irreversible neurological impairment in the early years of the disease.
Shuna Chen, Mingqin Zhu, Yulei Hao, Jiachun Feng, Ying Zhang   +4 more
doaj   +1 more source