Results 21 to 30 of about 482,314 (268)

Assessment of Barriers to Referral and Appointment Wait Times for the Evaluation of Spinal Muscular Atrophy (SMA): Findings from a Web-Based Physician Survey

Neurology and Therapy
Background Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by progressive muscle weakness and atrophy. Clinical trial data suggest early diagnosis and treatment are critical.
Mary A. Curry, Rosángel E. Cruz, Lisa T. Belter, Mary K. Schroth, Jill Jarecki   +4 more
doaj   +1 more source

Fatal Relapse of Myelodysplastic Syndrome in a Patient with HIV/Hepatitis C Coinfection Treated with Simeprevir/Sofosbuvir

Journal of the International Association of Providers of AIDS Care, 2017
Registrational studies and observational cohorts clearly suggest sustained virologic response (SVR) rates in HIV-/hepatitis C–coinfected patients are similar to monoinfected patients when utilizing interferon-free regimens, and this can be accomplished ...
Efemena Michael Diejomaoh MD, Joseph Clayton Gathe MD, FACP, FIDSA, Carl Craig Mayberry PA-C, John Benjamin Clemmons MD, Bernie Miguel MD, Alan Glombicki MD, Benjamin Daquioag MD   +6 more
doaj   +1 more source

Chemotherapy Curability in Leukemia, Lymphoma, Germ Cell Tumors and Gestational Malignancies: A Reflection of the Unique Physiology of Their Cells of Origin

Frontiers in Genetics, 2020
Cytotoxic DNA damaging chemotherapy brings clinical benefits in the treatment of many metastatic malignancies. However routine curative treatment remains restricted to a small number of malignancies including acute leukemia, high grade lymphoma, germ ...
Philip Savage
doaj   +1 more source

Controversies in the Design of Strategies for the Cure of HIV Infection

Pathogens, 2023
The cure for chronic human immunodeficiency virus (HIV) infections has been a goal pursued since the antiretroviral therapy that improved the clinical conditions of patients became available.
Alejandro de Gea-Grela, Santiago Moreno
doaj   +1 more source

Genomic Diversity and Clinical Variability in Pediatric Primary Cutaneous Anaplastic Large Cell Lymphoma: A Case Series

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Primary cutaneous anaplastic large cell lymphoma (pcALCL) is a rare pediatric CD30‐positive T‐cell lymphoproliferative disorder with an excellent prognosis, but its genomic drivers are poorly defined. We report three children with skin‐limited disease demonstrating striking molecular heterogeneity, including NPM::ALK, NUP214::FRK, and a novel ...
Shoshana Greenberger, Efrat Bar‐Ilan, Victoria Marcu, Evgeny Grishin, Rinat Yacobi, Aviv Barzilai, Hana Golan, Assaf Arie Barg   +7 more
wiley   +1 more source

Are Fertility Preservation Procedures Before Gonadotoxic Therapy and Hematopoietic Stem Cell Transplantation Feasible and Safe in Very Young Children? A Retrospective Cohort Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Fertility preservation (FP) is increasingly integrated into the care of pediatric patients exposed to gonadotoxic therapy or conditioning for hematopoietic stem cell transplantation (HSCT), yet perioperative data in infants and toddlers remain scarce.
Kerstin Saalabian, Yannick Braun, Till‐Martin Theilen, Anke Barnbrock, Theresa Rohm, Fabienne Hammant, Nicole Sänger, Aynura Abbasova‐Semiz, Jan‐Henning Klusmann, Thomas Klingebiel, Udo Rolle, Peter Bader, Henning C. Fiegel, Andrea Jarisch   +13 more
wiley   +1 more source

A qualitative, mixed-method approach to reaching consensus on function, fatigue, and fatigability outcomes in teens and adults living with spinal muscular atrophy

Orphanet Journal of Rare Diseases
Background Spinal muscular atrophy (SMA) is a neuromuscular disease caused by mutations in the survival motor neuron gene, SMN1. Loss of SMN1 function results in deficiency of the SMN protein leading to motor neuron death, muscle wasting, and progressive
Jacqueline Glascock, Lisa T. Belter, Meghan Moore Burk, Jessica J. Tingey, Mary A. Curry   +4 more
doaj   +1 more source

Central Nervous System Neuroblastoma, FOXR2‐Activated: A Pooled Analysis of Published Clinical Outcomes

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Central nervous system (CNS) neuroblastoma, FOXR2‐activated, is a recently recognized entity in the WHO CNS5 classification, defined by activation of the FOXR2 transcription factor and unique histopathological features. This review synthesizes available literature and pooled clinical data, providing insight into demographics ...
Sudarshawn Damodharan, Mohamed S. Abdelbaki   +1 more
wiley   +1 more source

Reseña de Masa Goro. La crianza de “personas verdaderas” entre los macuna del bajo Apaporis”.

Mundo Amazónico, 2018
Masa Goro. La crianza de “personas verdaderas” entre los macuna del bajo Apaporis”. Dany Mahecha, 2015.
Salima Cure
doaj   +1 more source

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