Results 81 to 90 of about 7,599,301 (308)

Therapeutic Apheresis in Nigeria: A Multi‐Center Summary of Abstracts From the Inaugural Nigerian Society for Apheresis Scientific Meeting

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Background Therapeutic apheresis (TA) is an established treatment modality for hematologic, neurologic, and immunologic disorders, yet access remains severely limited in sub‐Saharan Africa. Donor apheresis, including platelet apheresis collection from healthy donors, represents an important complementary modality supporting blood product ...
Nosa Bazuaye, Chimezie Okwuonu, Evelyn Idam, Folasade Adelekan‐Popoola, Shakirat Gold‐Olufadi, Olufunto Olufela Kalejaiye, Abdullateef Ogala, Zinnah Aloziem, Blossom Amamchukwu, Patience Elimia, Mkpoikanabasi Obot‐Obot, Henry Mbakwe, Praises Maduagwuna, Olatunji Akinrodoye, Chiamaka Anyanwu, Benjamin Anyanwu, Adedamola Akinsiku, Oluwatoyin A. Akinsiku, Muzamil O. Hassan, Fatiu A. Arogundade, Olalekan E. Ojo, Homayemem Weli, Jesupelumi Adenihun, Aderemi Adelaja, Ebun Bamgboye, Olalekan Olatise, Ade E. Faponle, Ayoola Odeyemi, Michael Muoka, Stephen Asaolu, Qudrat Busari, Blessing Dawudu, Michael Naiyeju, Rasheed A. Balogun   +33 more
wiley   +1 more source

Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan

Frontiers in Neurology
Background and purposeGuillain-Barré syndrome (GBS) is an autoimmune neurological disorder characterized by muscle weakness. In clinical trials, treatment benefit and disease severity are typically measured using clinician-reported outcome measures like ...
Antoine Regnault, Angély Loubert, Stéphane Quéré, Qun Lin, Glen Frick, Hirokazu Ishida, Yuko Abeta, Helene Chevrou-Severac   +7 more
doaj   +1 more source

Forecasting the Dialysis Burden in Japan: Validation‐Based Projections of Prevalence and Incidence Through 2050

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Background Japan has one of the highest dialysis prevalence rates worldwide and a shrinking, aging population. Whether dialysis burden has entered a sustained post‐peak phase or whether recent declines partly reflect pandemic‐related disruptions remains uncertain.
Hatice Şahin, Berrak Itır Aylı, Nüket Paksoy   +2 more
wiley   +1 more source

Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency

Communications Medicine
Background Multiple Sulfatase Deficiency (MSD) is a rare inherited lysosomal storage disorder characterized by loss of function mutations in the SUMF1 gene that manifests as a severe pediatric neurological disease.
Maximiliano Presa, Rachel M. Bailey, Somdatta Ray, Lauren Bailey, Saurabh Tata, Tara Murphy, Pierre-Alexandre Piec, Harold Combs, Steven J. Gray, Cathleen Lutz   +9 more
doaj   +1 more source

Organoids in pediatric cancer research

FEBS Letters, EarlyView.
Organoid technology has revolutionized cancer research, yet its application in pediatric oncology remains limited. Recent advances have enabled the development of pediatric tumor organoids, offering new insights into disease biology, treatment response, and interactions with the tumor microenvironment.
Carla Ríos Arceo, Jarno Drost
wiley   +1 more source

An Easy Guide to Rare Diseases in Ireland: A Resource for the Media and the General Public

, 2020
This ‘Easy Guide’ has been produced to increase awareness about the challenges of living with a rare disease and to highlight the priorities that need to be included in the forthcoming National Plan for Rare Diseases (NPRD) in Ireland. The aims of this
Task Force on Rare Diseases
core  

Rare diseases [PDF]

Thorax, 1999
C M ROBERTS;, A E TATTERSFIELD, R M DU BOIS   +2 more
openaire   +2 more sources

From mice to humans—divergent strategies for intestinal homeostasis and regeneration

FEBS Letters, EarlyView.
Recent advances such as organoid genome editing, xenotransplantation, imaging, and whole‐genome sequencing have enabled direct studies of human intestinal stem cells (ISCs). These studies reveal species‐specific features, including slower ISC proliferation, distinct injury responses, slower somatic mutation accumulation in humans, and an inverse ...
Keiko Ishikawa, Shinya Sugimoto, Toshiro Sato   +2 more
wiley   +1 more source

Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice

Molecular Therapy: Methods & Clinical Development
Pompe disease (PD) is a multisystemic progressive disease caused by acid-alpha glucosidase (GAA) deficiency. Patients display a spectrum of phenotypes ranging from the severe, rapidly progressive infantile-onset PD (IOPD) form to the slower progressing ...
Kelly George, Raquel Riley, Shan Zhou, Elizabeth Allen, Laurie Smith, Elena Kistanova, Sofia Kinton, Lilu Guo, Dinesh Bangari, Ayman Ismail, Jayaprakash Thummapudi, Fawzi Boumezbeur, Erwan Selingue, Dorothée Tamarelle, Cecile Capdevila, Tuan-Minh Do, Dominique Lesuisse, Nina Leksa, Pablo Sardi, Arjan van der Flier   +19 more
doaj   +1 more source

Lost in the Vaso‐Occlusion: A Patient's Abdominal Pain Returns With a Vengeance

Pediatric Blood &Cancer, EarlyView.
Dunia Hatabah, Martha E. Stewart, Nathan Maassel, James P. Stevens, Claudia R. Morris, Chris A. Rees   +5 more
wiley   +1 more source