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1 . Academic Journal
Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy
저자
by
Mayhew, Anna G
;
Moat, Dionne
;
McDermott, Michael P.
;
Eagle, Michelle
;
Griggs, Robert C.
, et al.
소스
In
Neuromuscular Disorders
June 2022 32(6):460-467
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7 . Academic Journal
The 100-meter timed test: Normative data in healthy males and comparative pilot outcome data for use in Duchenne muscular dystrophy clinical trials
저자
by
Alfano, Lindsay N.
;
Miller, Natalie F.
;
Berry, Katherine M.
;
Yin, Han
;
Rolf, Kimberly E.
, et al.
소스
In
Neuromuscular Disorders
May 2017 27(5):452-457
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7 . Academic Journal
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
저자
by
Mendell, Jerry R.
;
Sahenk, Zarife
;
Al-Zaidy, Samiah
;
Rodino-Klapac, Louise R.
;
Lowes, Linda P.
, et al.
소스
In
Molecular Therapy
5 April 2017 25(4):870-879
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7 . Academic Journal
Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.
저자
by
P. Lowes, Linda
;
Alfano, Lindsay N.
;
Iammarino, Megan A.
;
Reash, Natalie F.
;
Giblin, Kathryn
, et al.
소스
PLoS ONE
. 5/16/2024, Vol. 19 Issue 5, p1-13. 13p.
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7 . Academic Journal
Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study.
저자
by
Iammarino, Megan A.
;
Alfano, Lindsay N.
;
Reash, Natalie F.
;
Sabo, Brenna
;
Conroy, Sara
, et al.
소스
PLoS ONE
. 3/19/2024, Vol. 19 Issue 3, p1-16. 16p.
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7 . Academic Journal
Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
저자
by
Mendell, Jerry R.
;
Sahenk, Zarife
;
Lehman, Kelly J.
;
Lowes, Linda P.
;
Reash, Natalie F.
, et al.
소스
Muscle & Nerve
. Jan2024, Vol. 69 Issue 1, p93-98. 6p.
Full Text (Wiley)
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7 . Academic Journal
Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network
저자
by
Connolly, Anne M.
;
Florence, Julaine M.
;
Cradock, Mary M.
;
Malkus, Elizabeth C.
;
Schierbecker, Jeanine R.
, et al.
소스
In
Neuromuscular Disorders
July 2013 23(7):529-539
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7 . Periodical
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results
저자
by
Mendell, Jerry R.
;
Pozsgai, Eric R.
;
Lewis, Sarah
;
Griffin, Danielle A.
;
Lowes, Linda P.
, et al.
소스
Nature Medicine; January 2024, Vol. 30 Issue: 1 p199-206, 8p
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7 . Academic Journal
WiTNNess: An international natural history study of infantile‐onset TNNT1 myopathy.
저자
by
Strauss, Kevin A.
;
Carson, Vincent J.
;
Bolettieri, Emilienne
;
Everett, Mariah
;
Bollinger, Ashton
, et al.
소스
Annals of Clinical & Translational Neurology
. Nov2023, Vol. 10 Issue 11, p1972-1984. 13p.
Open Access (Wiley)
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7 . Academic Journal
266th ENMC International Workshop: Remote delivery of clinical care and validation of remote clinical outcome assessments in neuromuscular disorders: A response to COVID-19 and proactive planning for the future. Hoofddorp, The Netherlands, 1–3 April 2022
저자
by
Alfano, Lindsay N.
;
James, Meredith K.
;
Ramdharry, Gita M.
;
Lowes, Linda P.
.
소스
Neuromuscular Disorders
. Apr2023, Vol. 33 Issue 4, p339-348. 10p.
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