Modeling disease trajectory in Duchenne muscular dystrophy.


Journal

Neurology
ISSN: 1526-632X
Titre abrégé: Neurology
Pays: United States
ID NLM: 0401060

Informations de publication

Date de publication:
14 04 2020
Historique:
received: 02 07 2019
accepted: 17 10 2019
pubmed: 19 3 2020
medline: 14 8 2020
entrez: 19 3 2020
Statut: ppublish

Résumé

To quantify disease progression in individuals with Duchenne muscular dystrophy (DMD) using magnetic resonance biomarkers of leg muscles. MRI and magnetic resonance spectroscopy (MRS) biomarkers were acquired from 104 participants with DMD and 51 healthy controls using a prospective observational study design with patients with DMD followed up yearly for up to 6 years. Fat fractions (FFs) in vastus lateralis and soleus muscles were determined with MRS FF and MRI qT MRS FF and MRI qT NCT01484678.

Identifiants

pubmed: 32184340
pii: WNL.0000000000009244
doi: 10.1212/WNL.0000000000009244
pmc: PMC7251517
doi:

Substances chimiques

Adrenal Cortex Hormones 0
Biomarkers 0

Banques de données

ClinicalTrials.gov
['NCT01484678']

Types de publication

Journal Article Research Support, N.I.H., Extramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

e1622-e1633

Subventions

Organisme : NIAMS NIH HHS
ID : R01 AR065943
Pays : United States
Organisme : NIH HHS
ID : S10 OD021701
Pays : United States
Organisme : NIH HHS
ID : S10 OD018224
Pays : United States
Organisme : NIAMS NIH HHS
ID : U54 AR052646
Pays : United States
Organisme : NIAMS NIH HHS
ID : R01 AR056973
Pays : United States
Organisme : NIDA NIH HHS
ID : F30 DA033094
Pays : United States
Organisme : NIMH NIH HHS
ID : R25 MH101076
Pays : United States
Organisme : NHLBI NIH HHS
ID : T32 HL134621
Pays : United States

Informations de copyright

© 2020 American Academy of Neurology.

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Auteurs

William D Rooney (WD)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR. rooneyw@ohsu.edu.

Yosef A Berlow (YA)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

William T Triplett (WT)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Sean C Forbes (SC)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Rebecca J Willcocks (RJ)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Dah-Jyuu Wang (DJ)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Ishu Arpan (I)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Harneet Arora (H)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Claudia Senesac (C)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Donovan J Lott (DJ)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Gihan Tennekoon (G)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Richard Finkel (R)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Barry S Russman (BS)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Erika L Finanger (EL)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Saptarshi Chakraborty (S)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Elliott O'Brien (E)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Brendan Moloney (B)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Alison Barnard (A)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

H Lee Sweeney (HL)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Michael J Daniels (MJ)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Glenn A Walter (GA)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

Krista Vandenborne (K)

From the Advanced Imaging Research Center (W.D.R., Y.A.B., I.A., E.O., B.M.), Department of Neurology (W.D.R., I.A., B.S.R., E.L.F.), Department of Biomedical Engineering (W.D.R.), Department of Behavioral Neuroscience (W.D.R., Y.A.B.), and Department of Pediatrics (B.S.R., E.L.F.), Oregon Health & Science University, Portland; Departments of Physical Therapy (W.T.T., S.C.F., R.J.W., H.A., C.S., D.J.L., K.V.), Statistics (S.C., M.J.D.), Physiology and Functional Genomics (A.B., G.A.W.), and Pharmacology & Therapeutics (H.L.S.), University of Florida, Gainesville; Department of Radiology (D.-J.W.) and Division of Neurology (G.T.), Children's Hospital of Philadelphia, PA; Department of Pediatrics (R.F.), Nemours Children's Hospital, Orlando, FL; and Shriners Hospital (B.S.R., E.L.F.), Portland, OR.

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