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Akademický článek

Lower limb muscle MRI fat fraction is a responsive outcome measure in CMT X1, 1B and 2A

Publikováno v: Annals of Clinical and Translational Neurology, Vol 11, Iss 3, Pp 607-617 (2024)

Abstract Objective With potential therapies for many forms of Charcot‐Marie‐Tooth disease (CMT), responsive outcome measures are urgently needed for clinical trials. Quantitative lower limb MRI demonstrated progressive calf intramuscular fat accu

Externí odkaz: https://doaj.org/article/b655160e70c64a5fac42d356158f7451

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Akademický článek

Patient-reported impact of symptoms in adrenoleukodystrophy (PRISM-ALD)

Autor: Anika Varma, Jennifer Weinstein, Jamison Seabury, Spencer Rosero, Nuran Dilek, John Heatwole, Charlotte Engebrecht, Shaweta Khosa, Kaitlin Chung, Asif Paker, Amy Woo, Gregory Brooks, Chan Beals, Rohan Gandhi, Chad Heatwole

Publikováno v: Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-22 (2024)

Abstract Background Adrenoleukodystrophy (ALD) is a multifaceted, X-linked, neurodegenerative disorder that comprises several clinical phenotypes. ALD affects patients through a variety of physical, emotional, social, and other disease-specific facto

Externí odkaz: https://doaj.org/article/b05f6dc5d6844e1eb9c2b11d419d422b

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Akademický článek

Patient reported impact of symptoms in amyotrophic lateral sclerosis (PRISM-ALS): A national, cross-sectional studyResearch in context

Autor: Christine Zizzi, Jamison Seabury, Spencer Rosero, Danae Alexandrou, Ellen Wagner, Jennifer S. Weinstein, Anika Varma, Nuran Dilek, John Heatwole, Joanne Wuu, James Caress, Richard Bedlack, Volkan Granit, Jeffrey M. Statland, Paul Mehta, Michael Benatar, Chad Heatwole

Publikováno v: EClinicalMedicine, Vol 55, Iss , Pp 101768- (2023)

Summary: Background: As novel therapeutic interventions are being developed and tested in the amyotrophic lateral sclerosis (ALS) population, there is a need to better understand the symptoms and issues that have the greatest impact on the lives of i

Externí odkaz: https://doaj.org/article/8588de4be3c14594b6b567d84d574240

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Patient-Reported Impact of Symptoms in Friedreich Ataxia

Autor: Jamison Seabury, Danae Alexandrou, Nuran Dilek, Brittany Cohen, John Heatwole, Jane Larkindale, David R. Lynch, Courtney Park, Spencer Rosero, Sub H. Subramony, Anika Varma, Ellen Wagner, Susan Walther, Jennifer Weinstein, McKenzie Wells, Christine Zizzi, Chad Heatwole

Publikováno v: Neurology

Background and ObjectivesTo determine the prevalence and relative importance of symptoms experienced by children and adults with Friedreich ataxia (FA) and to identify factors associated with a higher burden of disease.MethodsWe conducted qualitative

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a0df1e01df7c43883e5c21de29decc1b
https://doi.org/10.1212/wnl.0000000000201598

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The Huntington’s Disease Health Index: Initial Evaluation of a Disease-Specific Patient Reported Outcome Measure

Autor: Olivia S, Brumfield, Christine E, Zizzi, Nuran, Dilek, Danae G, Alexandrou, Alistair M, Glidden, Spencer, Rosero, Jennifer, Weinstein, Jamison, Seabury, Aaron, Kaat, Michael P, McDermott, E Ray, Dorsey, Chad R, Heatwole

Publikováno v: Journal of Huntington's Disease. 11:217-226

Background: When developed properly, disease-specific patient reported outcome measures have the potential to measure relevant changes in how a patient feels and functions in the context of a therapeutic trial. The Huntington’s Disease Health Index

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5f6af8e021fddf373e347c51091875a2
https://doi.org/10.3233/jhd-210506

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Patient-Reported Impact of Symptoms in Adrenoleukodystrophy (PRISM-ALD)

Background Adrenoleukodystrophy (ALD) is a multifaceted, x-linked, neurodegenerative disorder that affect patients through a variety of physical, emotional, social, and other disease-specific factors that collectively contribute to disease burden. Ad

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::0ff9e97c5ddb75955bb72f0c0212cf7d
https://doi.org/10.21203/rs.3.rs-2856459/v1

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