Detalhe da pesquisa
1.
Telemedicine Use, Comfort, and Perceived Effectiveness in the Spinal Muscular Atrophy Community.
Telemed J E Health
; 30(2): 536-544, 2024 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-37566530
2.
Assessing perspectives of disease burden and clinically meaningful changes using the Spinal Muscular Atrophy Health Index in adolescents and young adults.
Muscle Nerve
; 66(3): 276-281, 2022 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35616549
3.
Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophy.
Prenat Diagn
; 42(11): 1409-1419, 2022 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-36029101
4.
Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part study.
BMC Neurol
; 21(1): 143, 2021 Mar 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-33789607
5.
Awareness screening and referral patterns among pediatricians in the United States related to early clinical features of spinal muscular atrophy (SMA).
BMC Pediatr
; 21(1): 236, 2021 05 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-34001052
6.
Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders.
Clin Trials
; 15(2): 139-148, 2018 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-29475375
7.
An Evidence-Based, Community-Engaged Approach to Develop an Interactive Deliberation Tool for Pediatric Neuromuscular Trials.
J Genet Couns
; 27(2): 416-425, 2018 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-29260486
8.
A qualitative study of perceptions of meaningful change in spinal muscular atrophy.
BMC Neurol
; 17(1): 68, 2017 Apr 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28376816
9.
Correction to: Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part study.
BMC Neurol
; 21(1): 354, 2021 Sep 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-34517827
10.
The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models.
Hum Mol Genet
; 22(20): 4084-101, 2013 Oct 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-23736298
11.
Understanding the experiences and needs of individuals with Spinal Muscular Atrophy and their parents: a qualitative study.
BMC Neurol
; 15: 217, 2015 Oct 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-26499462
12.
Assessment of Barriers to Referral and Appointment Wait Times for the Evaluation of Spinal Muscular Atrophy (SMA): Findings from a Web-Based Physician Survey.
Neurol Ther
; 13(3): 583-598, 2024 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-38430355
13.
Evaluating Perceived Fatigue within an Adult Spinal Muscular Atrophy Population.
Neurol Ther
; 12(6): 2161-2175, 2023 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-37856000
14.
Effects of the COVID-19 Pandemic on SMA Screening and Care: Physician and Community Insights.
Neurol Ther
; 12(5): 1631-1647, 2023 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-37347432
15.
Identifying Biomarkers of Spinal Muscular Atrophy for Further Development.
J Neuromuscul Dis
; 10(5): 937-954, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37458045
16.
Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy.
Hum Mol Genet
; 19(3): 454-67, 2010 Feb 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-19897588
17.
Knowledge of genetic test results among caregivers and individuals with spinal muscular atrophy.
PLoS One
; 17(11): e0276756, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-36346824
18.
Clinical and Research Readiness for Spinal Muscular Atrophy: The Time Is Now for Knowledge Translation.
Phys Ther
; 102(10)2022 10 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35904447
19.
The Cure SMA Clinical Trial Experience Survey: A Study of Trial Participant Perspectives on Clinical Trial Management and Patient-Centric Management Practices.
Neurol Ther
; 11(3): 1167-1181, 2022 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-35635603
20.
"I have SMA, SMA doesn't have me": a qualitative snapshot into the challenges, successes, and quality of life of adolescents and young adults with SMA.
Orphanet J Rare Dis
; 16(1): 96, 2021 02 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-33618755