Results 41 to 50 of about 4,579,575 (247)

Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale

open access: yesOrphanet Journal of Rare Diseases, 2021
Background Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, genetic disease leading to impaired lysosomal function and neurodegeneration causing serious morbidity and shortened life expectancy.
Marc C. Patterson   +8 more
doaj   +1 more source

Retrospective Analysis of Donor Lymphocyte Infusions in Pediatric Patients With Mixed Chimerism After Hematopoietic Stem Cell Transplantation

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Background Allogeneic hematopoietic stem cell transplantation (alloHSCT) is an essential therapy for several malignant and nonmalignant diseases, but relapse and graft loss remain the principal threats to its success. Routine monitoring of chimerism and minimal residual disease (MRD) enables early detection of imminent recurrence and guides ...
Carmen Junk   +10 more
wiley   +1 more source

Economic and Treatment Burden of Chronic Low-Back Pain Patients Transitioning from Short-Acting to Long-Acting Opioids or Buprenorphine Buccal Film: US Claims Analysis

open access: yesPain and Therapy
Introduction This study aimed to assess economic burden and treatment characteristics of patients with chronic low-back pain (cLBP) transitioning from oral Schedule II (CII) short-acting opioids (SAO) to oral CII long-acting opioids (LAO) or Belbuca ...
Filip Stanicic   +2 more
doaj   +1 more source

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

open access: yesPediatric 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   +17 more
wiley   +1 more source

The Impact of the COVID‐19 Pandemic on Childhood Cancer Survival: A Population‐Based Assessment of Survival Patterns Between 2015 and 2023 in Germany

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Background Numerous international studies have reported declines in new cancer diagnoses, delayed diagnoses and disruptions in cancer treatment following the implementation of COVID‐19 pandemic public health measures, raising concerns that these effects may ultimately contribute to increased cancer mortality.
Friederike Erdmann   +8 more
wiley   +1 more source

Outcomes and Aging [PDF]

open access: yesPhysical Therapy, 2000
To the Editor: I am writing to you regarding the report on anterior cruciate ligament (ACL) rehabilitation by Fitzgerald et al and your subsequent discussion with Mark DeCarlo, James Irrgang, and Kevin Wilk in the February 2000 issue of the Journal. I read the report with great interest that is not only clinical in origin but also personal in origin,
openaire   +2 more sources

Development of the Near Vision Presbyopia Task-based Questionnaire for use in evaluating the impact of presbyopia

open access: yesJournal of Patient-Reported Outcomes, 2021
Background Presbyopia is a progressive condition that reduces the eye’s ability to focus on near objects with increasing age. After a systematic literature review identified no existing presbyopia-specific patient-reported outcome (PRO) instruments ...
Elaheh Shirneshan   +8 more
doaj   +1 more source

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

open access: yesTherapeutic 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   +33 more
wiley   +1 more source

Patient Experience of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): A Qualitative Exploration of Signs, Symptoms, and Health-Related Quality of Life Impacts

open access: yesNeurology and Therapy
Introduction Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare type of autoimmune neuropathy, characterized by signs of distal and proximal weakness of the upper and lower limbs, sensory dysfunction, absent or diminished tendon reflexes,
Anna Roberts   +9 more
doaj   +1 more source

Experience With Performing Rheocarna Therapy via the Single‐Needle Method for Treatment of Chronic Limb‐Threatening Ischemia

open access: yesTherapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Introduction This study investigated the safety and efficacy of single‐needle Rheocarna therapy for chronic limb‐threatening ischemia (CLTI) with wounds. Methods Six patients with CLTI involving ulcers unresponsive to revascularization underwent single‐needle Rheocarna treatment.
Yasutaka Yamauchi   +9 more
wiley   +1 more source

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