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- Mercuri, E., et al.
(författare)
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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- 2020
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
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Tidskriftsartikel (refereegranskat)abstract
- Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p <= 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
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| 2. |
- Denecke, R., et al.
(författare)
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Beamline 1511 at MAX II, capabilities and performance
- 1999
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Ingår i: Journal of Electron Spectroscopy and Related Phenomena. - 0368-2048. ; 101-103, s. 971-977
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Tidskriftsartikel (refereegranskat)abstract
- The new undulator beamline 1511 at MAX-lab, now under commissioning, has been optimized for X-ray emission and photoelectron spectroscopies. Using an SX-700 high flux monochromator the accessible photon energy range is from 90 eV to about 1500 eV. The performance of the undulator agrees very well with the specifications, as shown by measurements using a photodiode. The energy resolution of the monochromator has been checked using absorption measurements in a gas cell. It was found to meet the expectations and exceeds a resolving power of 10 000 at 244 eV. The photon flux as a function of energy has been recorded as well and gives a maximum flux of 3×1013 photons/s/100 mA/0.1% BW. Beamlines 1511 and 1411 will be the first synchrotron beamlines making use of a so-called beam waist phenomenon, known from laser physics. We show results of ray-tracing calculations to determine the ultimate spot size on the sample location. The endstations to be used at this new beamline and their capabilities will be discussed as an example of the future use of this facility.
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| 3. |
- Denecke, R, et al.
(författare)
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Beamline I511 at MAX II, capabilities and performance
- 1999
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Ingår i: JOURNAL OF ELECTRON SPECTROSCOPY AND RELATED PHENOMENA. - : ELSEVIER SCIENCE BV. - 0368-2048. ; 103, s. 971-977
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Tidskriftsartikel (övrigt vetenskapligt/konstnärligt)abstract
- The new undulator beamline I511 at MAX-lab, now under commissioning, has been optimized for X-ray emission and photoelectron spectroscopies. Using an SX-700 high flux monochromator the accessible photon energy range is from 90 eV to about 1500 eV. The per
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| 9. |
- Hasselstrom, J, et al.
(författare)
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Crystal-field splitting in coadsorbate systems: c(2X2) CO/K/Ni(100)
- 2000
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Ingår i: PHYSICAL REVIEW B. - : AMERICAN PHYSICAL SOC. - 0163-1829. ; 62:16, s. 11192-11196
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Tidskriftsartikel (refereegranskat)abstract
- It is demonstrated how the crystal field splitting (CFS) fine structure can be used to characterize a coadsorbate system. We have applied K 2p x-ray absorption spectroscopy (XAS) to the c(2X2) CO/K/Ni(100) system. The CFS fine structure is shown to be sen
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