42 research outputs found
ΠΠ±ΡΠ°Π±ΠΎΡΠΊΠ° ΠΎΠΏΡΠΈΡΠ΅ΡΠΊΠΈΡ ΠΈΠ·ΠΌΠ΅ΡΠ΅Π½ΠΈΠΉ ΡΡΠ°Π΅ΠΊΡΠΎΡΠΈΠΈ Π»Π΅ΡΠ°ΡΠ΅Π»ΡΠ½ΡΡ ΠΎΠ±ΡΠ΅ΠΊΡΠΎΠ²
Π Π°ΡΡΠΌΠΎΡΡΠ΅Π½Ρ ΠΌΠ΅ΡΠΎΠ΄Ρ ΡΡΠ°Π²Π½ΠΈΠ²Π°Π½ΠΈΡ ΡΠ³Π»ΠΎΠ²ΡΡ
ΠΈΠ·ΠΌΠ΅ΡΠ΅Π½ΠΈΠΉ ΠΏΠΎ ΡΠΏΠΎΡΠΎΠ±Ρ Π½Π°ΠΈΠΌΠ΅Π½ΡΡΠΈΡ
ΠΊΠ²Π°Π΄ΡΠ°ΡΠΎΠ²: ΠΌΠ΅ΡΠΎΠ΄ ΡΡΠ°Π²Π½ΠΈΠ²Π°Π½ΠΈΡ ΠΈΠ·ΠΌΠ΅ΡΠ΅Π½ΠΈΠΉ ΠΎΡΠ΄Π΅Π»ΡΠ½ΠΎ Π² ΠΊΠ°ΠΆΠ΄ΠΎΠΌ Π²ΡΠ΅ΠΌΠ΅Π½Π½ΠΎΠΌ ΡΠ΅ΡΠ΅Π½ΠΈΠΈ, ΠΏΡΠ΅Π΄ΠΏΠΎΠ»Π°Π³Π°ΡΡΠΈΠΉ Π½ΡΠ»Π΅Π²ΠΎΠ΅ ΠΌΠ°ΡΠ΅ΠΌΠ°ΡΠΈΡΠ΅ΡΠΊΠΎΠ΅ ΠΎΠΆΠΈΠ΄Π°Π½ΠΈΠ΅ ΡΠ»ΡΡΠ°ΠΉΠ½ΡΡ
ΠΎΡΠΈΠ±ΠΎΠΊ ΠΈΠ·ΠΌΠ΅ΡΠ΅Π½ΠΈΠΉ, ΠΈ ΠΌΠ΅ΡΠΎΠ΄ ΡΡΠ°Π²Π½ΠΈΠ²Π°Π½ΠΈΡ ΠΈΠ·Π±ΡΡΠΎΡΠ½ΡΡ
ΠΎΠΏΡΠΈΡΠ΅ΡΠΊΠΈΡ
ΠΈΠ·ΠΌΠ΅ΡΠ΅Π½ΠΈΠΉ Ρ ΠΏΠΎΠ΄Π°Π²Π»Π΅Π½ΠΈΠ΅ΠΌ ΠΈΡ
ΠΏΠΎΡΡΠΎΡΠ½Π½ΡΡ
ΡΠΈΡΡΠ΅ΠΌΠ°ΡΠΈΡΠ΅ΡΠΊΠΈΡ
ΠΎΡΠΈΠ±ΠΎΠΊ Π² ΠΏΡΠ΅Π΄ΠΏΠΎΠ»ΠΎΠΆΠ΅Π½ΠΈΠΈ Π·Π°ΡΠΎΡΠ΅Π½Π½ΠΎΡΡΠΈ ΠΈΠ·ΠΌΠ΅ΡΠ΅Π½ΠΈΠΉ ΠΊΠ°ΠΊ ΡΠ»ΡΡΠ°ΠΉΠ½ΡΠΌΠΈ, ΡΠ°ΠΊ ΠΈ Π½Π΅ΠΈΠ·Π²Π΅ΡΡΠ½ΡΠΌΠΈ ΠΏΠΎ Π²Π΅Π»ΠΈΡΠΈΠ½Π΅ ΠΈ Π·Π½Π°ΠΊΡ ΡΠΈΡΡΠ΅ΠΌΠ°ΡΠΈΡΠ΅ΡΠΊΠΈΠΌΠΈ ΠΏΠΎΠ³ΡΠ΅ΡΠ½ΠΎΡΡΡΠΌΠΈ.Π ΠΎΠ·Π³Π»ΡΠ½ΡΡΠΎ ΠΌΠ΅ΡΠΎΠ΄ΠΈ Π·ΡΡΠ²Π½ΡΠ²Π°Π½Π½Ρ ΠΊΡΡΠΎΠ²ΠΈΡ
Π²ΠΈΠΌΡΡΡΠ²Π°Π½Ρ Π·Π° ΡΠΏΠΎΡΠΎΠ±ΠΎΠΌ Π½Π°ΠΉΠΌΠ΅Π½ΡΠΈΡ
ΠΊΠ²Π°Π΄ΡΠ°ΡΡΠ²: ΠΌΠ΅ΡΠΎΠ΄ Π·ΡΡΠ²Π½ΡΠ²Π°Π½Π½Ρ Π²ΠΈΠΌΡΡΡΠ²Π°Π½Ρ ΠΎΠΊΡΠ΅ΠΌΠΎ Π² ΠΊΠΎΠΆΠ½ΠΎΠΌΡ ΡΠ°ΡΠΎΠ²ΠΎΠΌΡ ΡΠΎΠ·ΡΡΠ·Ρ, ΡΠΎ ΠΏΠ΅ΡΠ΅Π΄Π±Π°ΡΠ°Ρ Π½ΡΠ»ΡΠΎΠ²Π΅ ΠΌΠ°ΡΠ΅ΠΌΠ°ΡΠΈΡΠ½Π΅ ΠΎΡΡΠΊΡΠ²Π°Π½Π½Ρ Π²ΠΈΠΏΠ°Π΄ΠΊΠΎΠ²ΠΈΡ
ΠΏΠΎΡ
ΠΈΠ±ΠΎΠΊ Π²ΠΈΠΌΡΡΡΠ²Π°Π½Ρ, Ρ ΠΌΠ΅ΡΠΎΠ΄ Π·ΡΡΠ²Π½ΡΠ²Π°Π½Π½Ρ Π½Π°Π΄Π»ΠΈΡΠΊΠΎΠ²ΠΈΡ
ΠΎΠΏΡΠΈΡΠ½ΠΈΡ
Π²ΠΈΠΌΡΡΡΠ²Π°Π½Ρ ΡΠ· Π·Π°Π³Π»ΡΡΠ΅Π½Π½ΡΠΌ ΡΡ
ΠΏΠΎΡΡΡΠΉΠ½ΠΈΡ
ΡΠΈΡΡΠ΅ΠΌΠ°ΡΠΈΡΠ½ΠΈΡ
ΠΏΠΎΡ
ΠΈΠ±ΠΎΠΊ Ρ ΠΏΡΠΈΠΏΡΡΠ΅Π½Π½Ρ Π·Π°ΡΠΌΡΡΠ΅Π½ΠΎΡΡΡ Π²ΠΈΠΌΡΡΡΠ²Π°Π½Ρ ΡΠΊ Π²ΠΈΠΏΠ°Π΄ΠΊΠΎΠ²ΠΈΠΌΠΈ, ΡΠ°ΠΊ Ρ Π½Π΅Π²ΡΠ΄ΠΎΠΌΠΈΠΌΠΈ Π·Π° Π²Π΅Π»ΠΈΡΠΈΠ½ΠΎΡ ΡΠ° Π·Π½Π°ΠΊΠΎΠΌ ΡΠΈΡΡΠ΅ΠΌΠ°ΡΠΈΡΠ½ΠΈΠΌΠΈ ΠΏΠΎΡ
ΠΈΠ±ΠΊΠ°ΠΌΠΈ.The methods of equalizing angular measurements according to the method of least squares are examined: the method of equalizing measurements separately in each temporary section, that assumes the zero mathematical expectation of the random errors of measurements, and the method of equalizing excessive optical measurements with suppression of their constant systematic errors under the assumption of the obstruction of measurements by systematic errors both random and unknowns by value and sign
Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial
Introduction:
The North Star Ambulatory Assessment (NSAA) tool is a key instrument for measuring clinical outcomes in patients with Duchenne muscular dystrophy (DMD). To gain a better understanding of the longitudinal utility of the NSAA, we evaluated NSAA data from a phase II trial of 120 patients with DMD treated with domagrozumab or placebo.
Methods:
The NSAA exploratory analyses included assessment of individual skills gained/lost, total skills gained/lost, cumulative loss of function, and the impact of transient loss of function due to a temporary disability on NSAA total score (temporary zero score).
Results:
There was no significant difference in the total number of NSAA skills gained (mean 1.41 and 1.04, respectively; p = 0.3314) or lost (3.90 vs. 5.0; p = 0.0998) between domagrozumab- vs. placebo-treated patients at week 49. However, domagrozumab-treated patients were less likely to lose the ability to perform a NSAA item (hazard ratio 0.80, 95% confidence interval [CI]: 0.65β0.98, p = 0.029) over 48-weeks vs. placebo-treated patients. When temporary zero scores were changed to βnot obtainableβ (8 values from 7 patients), domagrozumab-treated patients scored higher on the NSAA total score versus placebo-treated patients (difference at week 49: 2.0, 95% CI: 0.1β3.9, p = 0.0359).
Conclusions:
These exploratory analyses reveal additional approaches to interpreting the NSAA data beyond just change in NSAA total score. These observations also highlight the importance of reporting items as βnot obtainableβ for a patient with a temporary/transient physical disability that impacts their ability to perform the NSAA test
Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab
Duchenne muscular dystrophy (DMD) is a progressive, neuromuscular disorder caused by mutations in the DMD gene that results in a lack of functional dystrophin protein. Herein, we report the use of quantitative magnetic resonance imaging (MRI) measures as biomarkers in the context of a multicenter phase 2, randomized, placebo-controlled clinical trial evaluating the myostatin inhibitor domagrozumab in ambulatory boys with DMD (nβ=β120 aged 6 toβ<β16Β years). MRI scans of the thigh to measure muscle volume, muscle volume index (MVI), fat fraction, and T2 relaxation time were obtained at baseline and at weeks 17, 33, 49, and 97 as per protocol. These quantitative MRI measurements appeared to be sensitive and objective biomarkers for evaluating disease progression, with significant changes observed in muscle volume, MVI, and T2 mapping measures over time. To further explore the utility of quantitative MRI measures as biomarkers to inform longer term functional changes in this cohort, a regression analysis was performed and demonstrated that muscle volume, MVI, T2 mapping measures, and fat fraction assessment were significantly correlated with longer term changes in four-stair climb times and North Star Ambulatory Assessment functional scores. Finally, less favorable baseline measures of MVI, fat fraction of the muscle bundle, and fat fraction of lean muscle were significant risk factors for loss of ambulation over a 2-year monitoring period. These analyses suggest that MRI can be a valuable tool for use in clinical trials and may help inform future functional changes in DMD.Trial registration: ClinicalTrials.gov identifier, NCT02310763; registered December 2014
Spinal muscular atrophy care in the COVID-19 pandemic era
peer reviewedThe Corona Virus Disease 2019 (COVID-19) pandemic has resulted in reorganization of healthcare settings affecting the delivery of clinical care to patients with spinal muscular atrophy (SMA). There is a concern that patients with SMA may be at increased risk of manifesting severe symptoms of COVID-19. Currently approved therapies for SMA improve survival and motor function, however, their delivery requires an increased exposure to the health system and a dedicated healthcare team. In this paper, we discuss consensus recommendations pertaining to care of SMA patients during the pandemic. We highlight that SMA treatments should not be perceived as elective. Decisions regarding the delay of treatments should be made with consideration of the potential risks of COVID-19 exposure and the risk of that delay. We emphasize the importance of collaborative treatment decisions between the patient, family, and health care provider, considering any geographic or institution-specific policies and precautions for COVID-19. This article is protected by copyright. All rights reserved. This article is protected by copyright. All rights reserved
Integration of a Generalized Ratio of Polynomials
This paper provides a closed-form solution to the indefinite integral of a ratio of generalized polynomials where the denominator polynomial is raised to the general order rβZ+. Such an integral arises in physics and engineering, the solution of which allows for closed-form analysis