Results 31 to 40 of about 9,380,370 (380)

NFL is a marker of treatment response in children with SMA treated with nusinersen

Journal of Neurology, 2019
BackgroundRecently, the anti-sense oligonucleotide drug nusinersen was approved for spinal muscular atrophy (SMA) and our aim was to find a response marker for this treatment.MethodsTwelve children with SMA type 1 and two copies of the SMN2 gene were ...
B. Olsson, L. Alberg, N. Cullen, E. Michael, L. Wahlgren, A. Kroksmark, K. Rostásy, K. Blennow, H. Zetterberg, M. Tulinius   +9 more
semanticscholar   +1 more source

Health, wellbeing and lived experiences of adults with SMA: a scoping systematic review

Orphanet Journal of Rare Diseases, 2020
Background Spinal muscular atrophy (SMA) is a neurodegenerative disease that has a substantial and multifaceted burden on affected adults. While advances in supportive care and therapies are rapidly reshaping the therapeutic environment, these efforts ...
Hamish W. Y. Wan, K. Carey, Arlene M. D'Silva, S. Vucic, M. Kiernan, Nadine A. Kasparian, M. Farrar   +6 more
semanticscholar   +1 more source

Cheminformatics-based identification of phosphorylated RET tyrosine kinase inhibitors for human cancer

Frontiers in Chemistry
BackgroundRearranged during transfection (RET), an oncogenic protein, is associated with various cancers, including non-small-cell lung cancer (NSCLC), papillary thyroid cancer (PTC), pancreatic cancer, medullary thyroid cancer (MTC), breast cancer, and ...
Md. Enamul Kabir Talukder, Md. Enamul Kabir Talukder, Md. Aktaruzzaman, Md. Aktaruzzaman, Noimul Hasan Siddiquee, Noimul Hasan Siddiquee, Sabrina Islam, Tanveer A. Wani, Hamad M. Alkahtani, Seema Zargar, Md. Obayed Raihan, Md. Mashiar Rahman, Sushil Pokhrel, Foysal Ahammad   +13 more
doaj   +1 more source

One Year of Newborn Screening for SMA – Results of a German Pilot Project

Journal of Neuromuscular Diseases, 2019
Objective: Spinal muscular atrophy (SMA) is the most common neurodegenerative disease in childhood. The study was conducted to assess the impact of early detection of SMA by newborn screening (NBS) on the clinical course of the disease.
K. Vill, H. Kölbel, O. Schwartz, A. Blaschek, B. Olgemöller, E. Harms, S. Burggraf, W. Röschinger, J. Durner, D. Gläser, U. Nennstiel, B. Wirth, U. Schara, Beate Jensen, M. Becker, K. Hohenfellner, W. Müller-Felber   +16 more
semanticscholar   +1 more source

Enhanced oral bioavailability of cannabidiol by flexible zein nanoparticles: in vitro and pharmacokinetic studies

Frontiers in Nutrition
IntroductionCannabidiol (CBD) has a variety of pharmacological effects including antiepileptic, antispasmodic, anxiolytic and anti-inflammatory among other pharmacological effects.
YingLan Nie, Yan Kong, Juan Peng, Jian Sun, Bin Fan   +4 more
doaj   +1 more source

Fluorescent markers in microscopy: photophysical characteristics and applications in cell biology

Acta Biologica Slovenica, 2016
In the fluorescence microscopy of biological specimens the structures in cells and tissues usually need to be labelled with various fluorescent markers.
Urban Bogataj, Jasna Štrus, Nada Žnidaršič, Marko Kreft   +3 more
doaj   +1 more source

Submillimeter Array multiline observations of the massive star-forming region IRAS 18089-1732 [PDF]

, 2004
Submillimeter Array (SMA) observations of the high-mass star-forming region IRAS 18089-1732 in the 1 mm and 850 $\mu$m band with 1 GHz bandwidth reveal a wealth of information.
Beuther, H., Ho, P. T. P., Hunter, T. R., Lim, J., Liu, S. -Y., Ohashi, N., Sollins, P., Sridharan, T. K., Su, Y. N., Zhang, Q., Zhao, J. -H.   +10 more
core   +1 more source

An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials

CNS Drugs, 2019
Treatment with nusinersen has demonstrated significant and clinically meaningful benefits in clinical trials in infants and children with spinal muscular atrophy (SMA).
B. Darras, M. Farrar, E. Mercuri, R. Finkel, R. Foster, S. Hughes, I. Bhan, W. Farwell, S. Gheuens   +8 more
semanticscholar   +1 more source

Exploring lipid diversity and minimalism to define membrane requirements for synthetic cells

FEBS Letters, EarlyView.
Designing the lipid membrane of synthetic cells is a complex task, in which its various roles (among them solute transport, membrane protein support, and self‐replication) should all be integrated. In this review, we report the latest top‐down and bottom‐up advances and discuss compatibility and complexity issues of current engineering approaches ...
Sergiy Gan, Victoria Scarpelli, Marten Exterkate   +2 more
wiley   +1 more source

A three-dimensional lattice-free agent-based model of intracellular ice formation and propagation and intercellular mechanics in liver tissues

Royal Society Open Science
A successful cryopreservation of tissues and organs is crucial for medical procedures and drug development acceleration. However, there are only a few instances of successful tissue cryopreservation.
Fatemeh Amiri, James D. Benson
doaj   +1 more source