Results 1 to 10 of about 145,417 (165)

A conceptual disease model for quality of life in mitochondrial disease [PDF]

Orphanet Journal of Rare Diseases, 2022
Background Previous studies in patients with a mitochondrial disease (MD) highlight the high prevalence of cognitive impairments, fatigue, depression, and a lower quality of life (QoL).
Kim F. E. van de Loo, Nander T. van Zeijl, José A. E. Custers, Mirian C. H. Janssen, Christianne M. Verhaak   +4 more
doaj   +7 more sources

A conceptual model of functional health literacy to improve chronic airway disease outcomes [PDF]

BMC Public Health, 2021
Background Current conceptual models of health literacy (HL) illustrate the link between HL and health outcomes. However, these models fail to recognize and integrate certain elements of disease management, health system factors, and socio-demographic ...
Iraj Poureslami, Noah Tregobov, Jessica Shum, Austin McMillan, Alizeh Akhtar, Saron Kassay, Kassandra Starnes, Maryam Mahjoob, J. Mark FitzGerald   +8 more
doaj   +4 more sources

Carer burden in rare inherited diseases: a literature review and conceptual model [PDF]

Orphanet Journal of Rare Diseases, 2022
Background Carers of people living with rare diseases report heavy burden and a plethora of unmet needs. A previous parental supportive care needs framework has described the needs of parents of children living with rare diseases, but it is not specific ...
Kerry Sandilands, Angela Williams, Angela J. Rylands   +2 more
doaj   +4 more sources

The Lived Experience of Patients Diagnosed with Vitiligo and a Preliminary Conceptual Disease Model: Insights from Patients, Patient Advocates, and Clinicians [PDF]

Dermatology and Therapy
Introduction Patients with vitiligo experience high disease and psychosocial burden, and dissatisfaction with current treatment options, highlighting a need to understand the disease from a patient perspective.
Khaled Ezzedine, Ahmed M. Soliman, Mary Kathleen Ladd, Stavonnie Patterson, Rohini Sen, Sarah Ofori, Karin S. Coyne, Robin Pokrzywinski   +7 more
doaj   +4 more sources

The patient experience of Wilson disease: a conceptual model based on qualitative research [PDF]

Orphanet Journal of Rare Diseases, 2021
Background Wilson disease (WD) is a rare disease wherein copper accumulates in tissues, leading to hepatic degeneration, neurological impairments, and psychiatric symptoms.
Stella Karantzoulis, Karli Heuer, Nicole Sparling, Megan Teynor   +3 more
doaj   +4 more sources

Developing a conceptual model of symptoms and impacts in progressive fibrosing interstitial lung disease to evaluate patient-reported outcome measures [PDF]

ERJ Open Research, 2022
Background An understanding of the experience of patients with progressive fibrosing interstitial lung disease (PF-ILD) is needed to select appropriate patient-reported outcome measures (PROMs) to evaluate treatment effect in clinical trials.
Marlies Wijsenbeek, Maria Molina-Molina, Olivier Chassany, John Fox, Liam Galvin, Klaus Geissler, Katherine M. Hammitt, Michael Kreuter, Teng Moua, Emily C. O'Brien, Ashley F. Slagle, Anna Krasnow, Matthew Reaney, Michael Baldwin, Natalia Male, Klaus B. Rohr, Jeff Swigris, Katerina Antoniou   +17 more
doaj   +2 more sources

Developing a Conceptual Disease Model of Patient Experiences and Identifying Patient-Reported Clinical Outcome Assessments for Use in Trials of Treatments for Focal Onset Seizures [PDF]

Neuropsychiatric Disease and Treatment, 2022
Dorothee Oberdhan,1 Elizabeth Bacci,2 Jennifer N Hill,3 Andrew Palsgrove,1 Asha Hareendran3 1Otsuka Pharmaceutical Development & Commercialization, Inc., Rockville, MD, USA; 2Evidera, Seattle, WA, USA; 3Evidera, Bethesda, MD, USACorrespondence: Dorothee ...
Oberdhan D, Bacci E, Hill JN, Palsgrove A, Hareendran A   +4 more
doaj   +2 more sources

A conceptual disease model for adult Pompe disease. [PDF]

Orphanet J Rare Dis, 2015
Studies in orphan diseases are, by nature, confronted with small patient populations, meaning that randomized controlled trials will have limited statistical power. In order to estimate the effectiveness of treatments in orphan diseases and extrapolate effects into the future, alternative models might be needed.
Kanters TA, Redekop WK, Rutten-Van Mölken MP, Kruijshaar ME, Güngör D, van der Ploeg AT, Hakkaart L.   +6 more
europepmc   +4 more sources

Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease.

PLoS ONE, 2022
BackgroundSickle cell disease (SCD) is a clinically heterogeneous disease with many acute and chronic complications driven by ongoing vaso-occlusion and hemolysis. It causes a disproportionate burden on Black and Hispanic communities.
Kate M Johnson, Boshen Jiao, M A Bender, Scott D Ramsey, Beth Devine, Anirban Basu   +5 more
doaj   +2 more sources

On a new conceptual mathematical model dealing the current novel coronavirus-19 infectious disease

Results in Physics, 2020
The present paper describes a three compartment mathematical model to study the transmission of the current infection due to the novel coronavirus (2019-nCoV or COVID-19). We investigate the aforesaid dynamical model by using Atangana, Baleanu and Caputo
Anwarud Din, Kamal Shah, Aly Seadawy, Hussam Alrabaiah, Dumitru Baleanu   +4 more
doaj   +2 more sources