Results 81 to 90 of about 2,414,789 (346)

Understanding Disease Heterogeneity and Patient Characteristics in Patients with Amyotrophic Lateral Sclerosis (ALS) [PDF]

, 2019
Background: Amytrophic lateral sclerosis (ALS) is a fatal neurologic disease that is projected to double in worldwide incidence in the next 20 years.
Mathew, Veena
core  

Mechanisms of parasite‐mediated disruption of brain vessels

FEBS Letters, EarlyView.
Parasites can affect the blood vessels of the brain, often causing serious neurological problems. This review explains how different parasites interact with and disrupt these vessels, what this means for brain health, and why these processes matter. Understanding these mechanisms may help us develop better ways to prevent or treat brain infections in ...
Leonor Loira, Sílvia Arroz‐Madeira, Cláudio A. Franco, Sara Silva Pereira   +3 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

Sporadic Medullary Microcarcinoma in a Young Patient - A Rare Case [PDF]

, 2010
Sporadic medullary microcarcinoma of thyroid is a rare disease detected usually in 0.15% of all thyroid malignancy. We report a case of sporadic medullary microcarcinoma (MMC) of thyroid in a 24 year old male presenting as solitary thyroid nodule.
D, Manjunath, K, Amita, M, Abhishek, S, Vijayshankar   +3 more
core   +1 more source

Subacute Sclerosing Panencephalitis of the Brainstem as a Clinical Entity. [PDF]

, 2017
Subacute sclerosing panencephalitis (SSPE) is a rare progressive neurological disorder of early adolescence caused by persistent infection of the measles virus, which remains prevalent worldwide despite an effective vaccine. SSPE is a devastating disease
Ciacci, Joseph D, Upadhyayula, Pavan S, Yang, Jason, Yue, John K   +3 more
core   +3 more sources

PICALM::MLLT10 translocated leukemia

FEBS Letters, EarlyView.
This comprehensive review of PICALM::MLLT10 translocated acute leukemia provides an in‐depth review of the structure and function of CALM, AF10, and the fusion oncoprotein (1). The multifaceted molecular mechanisms of oncogenesis, including nucleocytoplasmic shuttling (2), epigenetic modifications (3), and disruption of endocytosis (4), are then ...
John M. Cullen, Antonia C. Nakatsugawa, Natalie Barton, Henry Haines, Gary S. Stein, Janet L. Stein, Daniel S. Wechsler, Jessica L. Heath   +7 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

A Phase II Basket Trial of Dual Anti-CTLA-4 and Anti-PD-1 Blockade in Rare Tumors (DART SWOG 1609) in Patients with Nonpancreatic Neuroendocrine Tumors. [PDF]

, 2020
PurposeImmune checkpoint blockade has improved outcomes across tumor types; little is known about the efficacy of these agents in rare tumors. We report the results of the (nonpancreatic) neuroendocrine neoplasm cohort of SWOG S1609 dual anti-CTLA-4 and ...
Baghdadi, Tareq Al, Blanke, Charles D, Chae, Young Kwang, Chen, Helen X, Fontaine, Annette, Gatalica, Zoran, Giles, Francis J, Hansel, Donna E, Hayward, Jourdain, Kasi, Anup, Korn, W Michael, Kurzrock, Razelle, Matrana, Marc, Mayerson, Edward, McLeod, Christine, Othus, Megan, Patel, Sandip P, Plets, Melissa, Ryan, Christopher W, Shah, Manisha H, Sharon, Elad, Singh, Preet Paul   +21 more
core  

Macrophage Activation Syndrome as Onset of Systemic Lupus Erythematosus: A Case Report and a Review of the Literature [PDF]

, 2015
Macrophage activation syndrome (MAS) is a potentially fatal condition. It is a rare complication of several autoimmune disorders, including systemic lupus erythematosus (SLE) and systemic juvenile idiopathic arthritis (sJIA).
Didona, Dario, Feola, Aldo, Granata, Guido, Granata, Massimo, Stifano, Giuseppina   +4 more
core   +4 more sources

A Young Man with a Rare Disease and an Unhealthy Habit [PDF]

, 2021
John G Williams, Robert M. Reed
openalex   +1 more source