Laura Lee Johnson

Assessing tolerability of cancer therapeutics: A regulatory perspective from the FDA Oncology Center of Excellence

Vishal Bhatnagar, Laura Lee Johnson, Mallorie H. Fiero, Flora A. Mulkey et autres

Patient-reported symptoms and functioning are valuable to understanding the tolerability of cancer treatments in clinical trials. Assessment of key side effects, physical and role functioning, and overall side effect bother in trials may be helpful for benefit-risk evaluation and useful to communicate …

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High-dose extended-release calcifediol successfully treated advanced secondary hyperparathyroidism in an end-stage kidney disease patient: a case report

Akhtar Ashfaq, John Choe, Samir P. Tabash, Stephen A. Strugnell et autres

BACKGROUND: Hemodialysis (HD) patients lose renal CYP27B1 (cytochrome P450 25-hydroxyvitamin D-1α-hydroxylase) as kidney function declines causing low serum total 25-hydroxyvitamin D (25D) and 1,25-dihydroxyvitamin D (1,25D), and elevated intact parathyroid hormone (iPTH). Most require vitamin D hormone treatment which increases the risk …

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Extended-Release Calcifediol Normalized 1,25-Dihydroxyvitamin D and Prevented Progression of Secondary Hyperparathyroidism in Hemodialysis Patients in a Pilot Randomized Clinical Trial

Charles W. Bishop, Akhtar Ashfaq, John Choe, Stephen A. Strugnell et autres

Introduction: Serum concentrations of 25-hydroxyvitamin D (25D) and 1,25-dihydroxyvitamin D (1,25D) decline as chronic kidney disease (CKD) advances, becoming insufficient without effective vitamin D repletion and driving onset of secondary hyperparathyroidism (SHPT). Randomized controlled trials (RCTs) in non-dialysis CKD patients have established …

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High-dose extended-release calcifediol successfully treated advanced secondary hyperparathyroidism in an end-stage kidney disease patient: a case report

Akhtar Ashfaq, John Choe, Samir P. Tabash, Stephen A. Strugnell et autres

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The Ercc1−/Δ mouse model of XFE progeroid syndrome undergoes accelerated retinal degeneration

Akilavalli Narasimhan, Seok Hong Min, Laura Lee Johnson, Heidi Roehrich et autres

Abstract Age‐related macular degeneration (AMD) is a major cause of vision loss in older adults. AMD is caused by degeneration in the macula of the retina. The retina is the highest oxygen consuming tissue in our body and is prone to oxidative …

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Nystagmus in the B6(CG)Tyr(c-2J)/J Albino Mouse: A Functional and RNA-Seq Analysis

Laura Lee Johnson, Juan E. Abrahante, Linda K. McLoon

Purpose: Infantile nystagmus syndrome (INS) is a gaze-holding disorder characterized by conjugate, uncontrolled eye oscillations that can result in significant visual acuity loss. INS is often associated with albinism, but the mechanism is unclear. Albino mice have nystagmus; however, a pigmented mouse …

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OP87 A Roadmap for Increasing The Usefulness And Impact Of Patient Preference Studies In Health Technology Assessment (HTA)

Deborah Anne Marshall, Esther Wilhelmina de Bekker-Grob, Brett Hauber, Sebastian Heidenreich et autres

Introduction The number of patient preference studies in health has increased dramatically. There is growing use of patient preferences in a wide variety of contexts, including health technology assessment. Patient preference studies can help inform decision makers on the needs and priorities …

Nystagmus Associated With the Absence of MYOD Expression Across the Lifespan in Extraocular and Limb Muscles

Laura Lee Johnson, Sadie L. Hebert, Rachel B. Kueppers, Linda K. McLoon

Purpose: The extraocular muscles (EOMs) undergo significant levels of continuous myonuclear turnover and myofiber remodeling throughout life, in contrast to limb skeletal muscles. Activation of the myogenic pathway in muscle precursor cells is controlled by myogenic transcription factors, such as MYOD. Limb …

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Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials-Innovative Medicines Initiative (SISAQOL-IMI): stakeholder views, objectives, and procedures

Madeline Lee Pe, Ahu Alanya, Ragnhild Sørum Falk, Cecilie Delphin Amdal et autres

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An Interview With the Food and Drug Administration About Draft Patient-Focused Drug Development Guidance 3: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments

Elisabeth M. Oehrlein, R. L. Bent, Fraser D. Bocell, Lili Garrard et autres

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Parasites, Infections, and Inoculation in Synthetic Minimal Cells

Brock Cash, Nathaniel J. Gaut, Christopher Deich, Laura Lee Johnson et autres

Synthetic minimal cells provide a controllable and engineerable model for biological processes. While much simpler than any live natural cell, synthetic cells offer a chassis for investigating the chemical foundations of key biological processes. Herein, we show a synthetic cell system with …

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Parasites, infections and inoculation in synthetic minimal cells

Brock Cash, Nathaniel J. Gaut, Christopher Deich, Laura Lee Johnson et autres

Abstract Synthetic minimal cells provide a controllable and engineerable model for an increasing amount of biological processes. While much simpler than any live natural cell, synthetic cells offer a chassis for investigating the foundations of key biological processes. Here we show a …

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