Results 61 to 70 of about 8,011,267 (345)

Neurochemical markers in CSF of adolescent and adult SMA patients undergoing nusinersen treatment

open access: yesTherapeutic Advances in Neurological Disorders, 2019
Background: There is limited information on neurochemical markers being used to support and monitor the affection of motoneurons in patients with spinal muscular atrophy (SMA).
C. Wurster   +14 more
semanticscholar   +1 more source

The immunological interface: dendritic cells as key regulators in metabolic dysfunction‐associated steatotic liver disease

open access: yesFEBS Letters, EarlyView.
Metabolic dysfunction‐associated steatotic liver disease (MASLD) affects nearly one‐third of the global population and poses a significant risk of progression to cirrhosis or liver cancer. Here, we discuss the roles of hepatic dendritic cell subtypes in MASLD, highlighting their distinct contributions to disease initiation and progression, and their ...
Camilla Klaimi   +3 more
wiley   +1 more source

The clinical landscape for SMA in a new therapeutic era

open access: yesGene Therapy, 2017
Despite significant advances in basic research, the treatment of degenerative diseases of the nervous system remains one of the greatest challenges for translational medicine.
K. Talbot, E. Tizzano
semanticscholar   +1 more source

Insights into PI3K/AKT signaling in B cell development and chronic lymphocytic leukemia

open access: yesFEBS Letters, EarlyView.
This Review explores how the phosphoinositide 3‐kinase and protein kinase B pathway shapes B cell development and drives chronic lymphocytic leukemia, a common blood cancer. It examines how signaling levels affect disease progression, addresses treatment challenges, and introduces novel experimental strategies to improve therapies and patient outcomes.
Maike Buchner
wiley   +1 more source

Specific inhibition of myostatin activation is beneficial in mouse models of SMA therapy

open access: yesHuman Molecular Genetics, 2018
Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by loss of &agr;‐motor neurons, leading to profound skeletal muscle atrophy. Patients also suffer from decreased bone mineral density and increased fracture risk.
K. Long   +11 more
semanticscholar   +1 more source

SMA - TREATMENT [PDF]

open access: yesNeuromuscular Disorders, 2021
Roy, S.   +12 more
openaire   +4 more sources

Taurine promotes glucagon‐like peptide‐1 secretion in enteroendocrine L cells

open access: yesFEBS Letters, EarlyView.
Taurine, a sulfur‐containing amino acid, is likely taken up by enteroendocrine L cells via the taurine transporter. This process increases the levels of cytosolic ATP. The increase in intracellular Ca2+ concentrations and glucagon‐like peptide‐1 secretion through membrane depolarization is caused by the closure of ATP‐sensitive potassium channels ...
Yuri Osuga   +6 more
wiley   +1 more source

Development of multi-pitch tool path in computer-controlled optical surfacing processes

open access: yesJournal of the European Optical Society-Rapid Publications, 2017
Background Tool path in computer-controlled optical surfacing (CCOS) processes has a great effect on middle spatial frequency error in terms of residual ripples.
Jing Hou, Defeng Liao, Hongxiang Wang
doaj   +1 more source

Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA).

open access: yesJournal of Medicinal Chemistry, 2018
Spinal muscular atrophy (SMA), a rare neuromuscular disorder, is the leading genetic cause of death in infants and toddlers. SMA is caused by the deletion or a loss of function mutation of the survival motor neuron 1 (SMN1) gene.
Atwood K Cheung   +32 more
semanticscholar   +1 more source

Making tau amyloid models in vitro: a crucial and underestimated challenge

open access: yesFEBS Letters, EarlyView.
This review highlights the challenges of producing in vitro amyloid assemblies of the tau protein. We review how accurately the existing protocols mimic tau deposits found in the brain of patients affected with tauopathies. We discuss the important properties that should be considered when forming amyloids and the benchmarks that should be used to ...
Julien Broc, Clara Piersson, Yann Fichou
wiley   +1 more source

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