Results 181 to 190 of about 46,815 (285)

HOPE Kids 2: Phase 3, Randomized Trial of Voxelotor in Children With SCD and Conditional Cerebral Blood Flow Velocities

American Journal of Hematology, EarlyView.
ABSTRACT Children with SCD have elevated stroke risk, correlated with cerebral blood flow velocity (CBFV). HOPE Kids 2 was a phase 3, multicenter, double‐blind, placebo‐controlled trial evaluating the effect of voxelotor on CBFV. Participants aged 2 to < 15 years with SCD (HbSS/HbSβ0) and conditional CBFV (170 to < 200 cm/s) were randomized 1:1 to ...
Halima Bello‐Manga, Shehu Umar Abdullahi, Hoda Hassab, Biobele Brown, Videlis Nduba, Jessie Githanga, Adeseye Michael Akinsete, Amal El‐Beshlawy, Diana Hanna, Catherine Segbefia, Iheanyi Okpala, Azza Tantawy, Bernhards Ogutu, Venée N. Tubman, Vivian Paintsil, Yasser Wali, Mohammed Essa, Paul Telfer, Sandy Dixon, Anne Hickman, Jennifer F. Doss, Mark W. Davis, Jeremy Rupon, Fenella J. Kirkham, R. Clark Brown, on behalf of the HOPE Kids 2 Investigators, Amal El‐Beshlawy, Mohamed Badr, Diana Hanna*, Hoda Hassab, Azza Tantawy, Vivian Paintsil, Catherine Segbefia, Raffaella Colombatti, Daniela Cuzzubbo, Silverio Perrotta, Jessie Githanga, Videlis Nduba, Bernhards Ogutu, Shehu Umar Abdullahi, Adeseye Michael Akinsete, Halima Bello‐Manga, Biobele Brown, Iheanyi Okpala, Yasser Wali, Mohammed Essa, Banu Kaya, Paul Telfer, Ofelia Alvarez, Natasha Archer, Shayla Bergmann, R Clark Brown, Andrew Campbell, Carlton Dampier*, Richard Hurwitz, Erin Jackson, Janet Kwiatkowski, Melissa M. McNaull*, Venée N. Tubman   +58 more
wiley   +1 more source

A digital archive reveals how a funding agency cooperated with academics to support the nascent field of genomics. [PDF]

Nat Commun
Hong SS, Utz Z, Hosseini M, Zanchettin C, Tejedor Navarro H, Holmes K, Wetterstrand KA, Bates SA, Nunes Amaral LA, Donohue CR, Stoeger T.   +10 more
europepmc   +1 more source

Navigating Asparaginase Treatment for Patients With Acute Lymphoblastic Leukemia: US Consensus Panel Recommendations on the Role of Multidisciplinary Care

American Journal of Hematology, EarlyView.
ABSTRACT Asparaginase is a critical treatment component for patients with acute lymphoblastic leukemia/lymphoblastic lymphoma (ALL/LBL). However, the successful delivery of asparaginase‐based therapy remains challenging across care settings due to its complex administration, distinct toxicity profile, prolonged treatment duration, and the need for ...
Amir Ali, Grace Elsey, Kayla Fulginiti, Zanette Kanani‐Bradley, Holly R. Kubaney, Sandra Kurtin, Theresa Nguyen, Jennifer Turchina, Charlotte B. Wagner   +8 more
wiley   +1 more source

Intra-articular synovial arteriovenous malformation: A case report study. [PDF]

Radiol Case Rep
Almuhanna M, Alhamad F, Almulhim M, Almaimani M, Aldraihem A, Aljabr A.   +5 more
europepmc   +1 more source

Phase II Study of Posttransplant Cyclophosphamide‐Based Graft‐Versus‐Host Disease Prophylaxis After HLA‐Mismatched Unrelated Donor Reduced Intensity Transplantation: Results From the ACCESS Trial Expansion Cohort

American Journal of Hematology, EarlyView.
ABSTRACT Posttransplant cyclophosphamide (PTCy) to prevent graft‐versus‐host disease (GVHD) improves outcomes in recipients of HLA mismatched unrelated donor (MMUD) allogeneic hematopoietic cell transplantation (allo HCT). Outcomes of MMUD HCT using PTCy in patients requiring reduced intensity or non‐myeloablative conditioning (RIC/NMA) are not well ...
Brian C. Shaffer, Monzr M. Al Malki, Antonio Martin Jimenez Jimenez, Joseph Stanek, Stephanie Bo‐Subait, Brent R. Logan, Jianqun Kou, Erin Leckrone, Heather E. Stefanski, Stephen R. Spellman, Craig Malmberg, Medhat Askar, Rachel Cusatis, Dipenkumar Modi, Farhad Khimani, Mehdi Hamadani, Martin Maiers, Rachel Cook, Karen Ballen, Javier Bolaños‐Meade, Uttam Rao, Jordan Milner, Ramzi Abboud, Katarzyna Jamieson, George Carrum, Bhagirathbhai Dholaria, William J. Hogan, Ran Reshef, Satyajit Kosuri, Alison Loren, Karilyn Larkin, Sally Arai, Muna Qayed, Sung Won Choi, Larisa Broglie, Jeffery J. Auletta, Bronwen E. Shaw, Steven M. Devine, Mahasweta Gooptu   +38 more
wiley   +1 more source

Gratitude in ID and a Challenge. [PDF]

Open Forum Infect Dis
Graf PJ, Hartzell JD.
europepmc   +1 more source

Accidents in the Chemical Industry: A Scoping Review Focused on Causal Factors

American Journal of Industrial Medicine, EarlyView.
ABSTRACT Background Although occupational safety in the chemical industry is an area of ongoing research worldwide, chemical incidents continue to occur, indicating the need to review the causes, assist in safety management, and identify problems in the quality of reporting. Methods A systematic search of studies published in English between January 1,
Željka Babić, Emin Veledar, Zrinka Franić, Franka Šakić, Mojca Dobaja Borak, Lucija Šarc, Jelena Macan, Miran Brvar   +7 more
wiley   +1 more source

In the footsteps of giants: a tale of legacy and discovery in medicine

Journal of Evidence-Based Healthcare
Mateus Maggitti-Bezerril, Bruno Bezerril Andrade   +1 more
doaj   +1 more source

Ocular and Systemic Findings in COL2A1 and COL11A1 Stickler Syndrome

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Stickler syndrome is most commonly caused by variants in COL2A1 and COL11A1 genes. The purpose of this study was to describe genetic variants and phenotypes in COL2A1 and COL11A1 Stickler syndrome. We performed a retrospective genotype–phenotype evaluation of COL2A1 and COL11A1 Stickler syndrome subjects. Thirty‐two subjects with COL2A1 and 13
Aileen G. MacLachlan, Jessica A. Kraker, Denise J. Morgan, Lisa A. Schimmenti, M. Elizabeth Hartnett, Eileen S. Hwang   +5 more
wiley   +1 more source

Innovation breakthrough in the Alzheimer's disease pharmaceutical industry. [PDF]

NPJ Drug Discov
Cheng H, Kang L, Xu Y, Tanzi RE, Zhang C, Wang C.   +5 more
europepmc   +1 more source