Results 31 to 40 of about 1,093,681 (262)

Feasibility and Safety of Somato‐Cognitive Coordination Therapy for Cerebellar Ataxia Following Pediatric Brain Tumor Treatment

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Cerebellar ataxia after pediatric brain tumor treatment can cause persistent gait, balance, and speech impairment, yet no established rehabilitation strategy exists. Somato‐cognitive coordination therapy (SCCT) is a virtual reality–guided intervention designed to promote sensorimotor integration through visually constrained reaching
Masanobu Takeuchi, Takashi Matsuzawa, Jun Yamanishi, Masahiro Yoshitomi, Junji Ikeda, Shin‐Ichi Tsujimoto, Yasuteru Yamaguchi, Norio Shiba, Dhroov Pathare, Ayumi Kato, Masahiko Hara   +10 more
wiley   +1 more source

Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6

Molecular Therapy: Methods & Clinical Development, 2023
Cone-rod dystrophy 6 (CORD6) is caused by gain-of-function mutations in the GUCY2D gene, which encodes retinal guanylate cyclase-1 (RetGC1). There are currently no treatments available for this autosomal dominant disease, which is characterized by severe,
Russell W. Mellen, Kaitlyn R. Calabro, K. Tyler McCullough, Sean M. Crosson, Alejandro de la Cova, Diego Fajardo, Emily Xu, Sanford L. Boye, Shannon E. Boye   +8 more
doaj   +1 more source

Prevalence of 21 Physician‐Defined Severe Toxicities Following Childhood Acute Lymphoblastic Leukemia Treatment: Australian Retrospective Cohort Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Acute lymphoblastic leukemia (ALL) is the most common pediatric cancer, with an overall survival now surpassing 90% in developed countries. However, treatments are not without adverse effects. In this study, we apply the severe toxicity‐free survival (STFS) framework to determine the prevalence of 21 physician‐defined severe ...
Lane Collier, Brittany Grantham, Camilla Grud Nielsen, Louise Lundgren, Liv Andrés‐Jensen, Kjeld Schmiegelow, Chris Selman, Gareth Ball, Elyse Passmore, David A. Elliott, Rachel Conyers   +10 more
wiley   +1 more source

Dynamics of BCMA expression in patients with relapsed/refractory multiple myeloma receiving BCMA-directed CAR-T therapy

Blood Cancer Journal
There is limited systemic data on the dynamics of BCMA-target antigen expression with BCMA CAR-T at relapse. We analyzed 76 patients receiving standard-of-care BCMA-directed CAR-T who underwent real-time BCMA expression evaluation at baseline (n = 50 ...
Masooma Shifa Rana, Sebastian Fernandez-Pol, Alexandria Jensen, Vanna Hovanky, Arash Velayati, Jean S. Oak, Oscar Silva, Erik Ames, Lori S. Muffly, Bita Sahaf, Sally Arai, Vanessa E. Kennedy, Sushma Bharadwaj, David J. Iberri, Michaela Liedtke, Yasodha Natkunam, Parveen Shiraz, Wen-Kai Weng, Melody Smith, Matthew J. Frank, Crystal L. Mackall, Saurabh Dahiya, Lekha Mikkilineni, David B. Miklos, Hitomi Hosoya, Surbhi Sidana   +25 more
doaj   +1 more source

Sustained Therapeutic Efficacy of Intravenous Plasminogen Concentrate in Pediatric Patients With Type 1 Plasminogen Deficiency: An Analysis of Dosing Parameters and Clinical Outcomes

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Type 1 plasminogen deficiency (PLGD‐1) is an ultra‐rare autosomal recessive disorder caused by variants in the PLG gene and affects approximately 1.6 individuals per million. The condition is characterized by decreased plasminogen levels and impaired function, resulting in fibrin‐rich lesions on mucous membranes throughout the body.
Charles Nakar, Karen Thibaudeau, Mary Mathias, Heather McDaniel, Madlen Reschke, Klaus Tenbrock, Joseph M. Parker, Amy Shapiro   +7 more
wiley   +1 more source

Cellular Therapy for Liver Disease [PDF]

Mayo Clinic Proceedings, 2014
Regenerative medicine is energizing and empowering basic science and has the potential to dramatically transform health care in the future. Given the remarkable intrinsic regenerative properties of the liver, as well as widespread adoption of regenerative strategies for liver disease (eg, liver transplant, partial hepatectomy, living donor transplant),
Robert C, Huebert, Jorge, Rakela
openaire   +2 more sources

Rational Use of Herbal Products in Pediatric Patients Treated With Anticancer Drugs in the European Union

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Introduction The use of herbal medical preparation (HMP) is rising among pediatric oncology patients, often to manage treatment‐related symptoms. Their effectiveness remains uncertain, and the risk of herb–drug interactions is underestimated.
Orianne Mahot, Mathieu Corvaisier, Laurence Spiesser‐Robelet, Mylène Duplan, Isabelle Pellier, Delphine Bourneau‐Martin, Séverine Derbré   +6 more
wiley   +1 more source

Steroid-Refractory Acute GVHD: Predictors and Outcomes

Advances in Hematology, 2011
Patients with steroid-resistant acute graft versus host disease (aGVHD) have a dismal prognosis, with mortality rates in excess of 90%. We sought to identify a subgroup of patients less likely to benefit from initial therapy with corticosteroids as well
Jason R. Westin, Rima M. Saliba, Marcos De Lima, Amin Alousi, Chitra Hosing, Muzaffar H. Qazilbash, Issa F. Khouri, Elizabeth J. Shpall, Paolo Anderlini, Gabriela Rondon, Borje S. Andersson, Richard Champlin, Daniel R. Couriel   +12 more
doaj   +1 more source

Improved Early Referral in Long‐Term Survivor Care With Institutional Standardized Practice Among Childhood Leukemia Survivors#

Pediatric Blood &Cancer, EarlyView.
ABSTRACT In 2018, the Texas Children's Cancer and Hematology Center Leukemia Program implemented a practice standard to support the transition from treatment to survivorship that includes shared, alternating care between leukemia and survivorship clinicians and a reminder to refer survivors to the long‐term survivor clinic (LTSC) 2 years after ...
Ji Yun Tark, Kayla L. Foster, Omar Shakeel, Mary C. Shapiro, Shawki L. Qasim, ZoAnn Dreyer, Eric S. Schafer, Ashley M. Butler, Austin L. Brown, Maria Monica Gramatges   +9 more
wiley   +1 more source

Personalized Zebrafish Models for Fusion‐Positive Pediatric Sarcomas

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Clinical sequencing efforts have revolutionized our approaches to categorizing pediatric cancers in real time. This has dramatically improved our ability to profile pediatric tumors, identify actionable vulnerabilities, and influence clinical care.
Lisa H. Hall, Emma N. Harrison, Genevieve C. Kendall   +2 more
wiley   +1 more source