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1.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-33789607
2.
Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy.
Health Qual Life Outcomes
; 19(1): 184, 2021 Jul 26.
Artículo
en Inglés
| MEDLINE | ID: mdl-34311756
3.
Use of Both Qualitative and Quantitative Methods to Estimate Meaningful Change Thresholds for Key Endpoints in Pediatric Asthma Trials.
Value Health
; 22(3): 340-347, 2019 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-30832972
4.
Assessing Asthma Symptoms in Adolescents and Adults: Qualitative Research Supporting Development of the Asthma Daily Symptom Diary.
Value Health
; 19(4): 440-50, 2016 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-27325336
5.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-34517827
6.
Stride Velocity 95th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy.
J Neuromuscul Dis
; 11(3): 701-714, 2024.
Artículo
en Inglés
| MEDLINE | ID: mdl-38640165
7.
The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.
Neurol Ther
; 13(1): 183-219, 2024 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-38190001
8.
Home-based video assessment of ease of movement for patients with Duchenne: Interviews with physical therapists to select movement tasks.
Physiother Res Int
; : e1993, 2023 Jan 30.
Artículo
en Inglés
| MEDLINE | ID: mdl-36718112
9.
A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale.
J Neuromuscul Dis
; 10(6): 1093-1109, 2023.
Artículo
en Inglés
| MEDLINE | ID: mdl-37742658
10.
Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA).
J Neurol
; 270(5): 2531-2546, 2023 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-36735057
11.
A Patient-Centered Conceptual Model of Symptoms and Their Impact in Early Parkinson's Disease: A Qualitative Study.
J Parkinsons Dis
; 12(1): 137-151, 2022.
Artículo
en Inglés
| MEDLINE | ID: mdl-34657850
12.
Development of the SMA independence scale-upper limb module (SMAIS-ULM): A novel scale for individuals with Type 2 and non-ambulant Type 3 SMA.
J Neurol Sci
; 432: 120059, 2022 Jan 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-34896922
13.
Asthma Daytime Symptom Diary (ADSD) and Asthma Nighttime Symptom Diary (ANSD): Measurement Properties of Novel Patient-Reported Symptom Measures.
J Allergy Clin Immunol Pract
; 10(5): 1249-1259, 2022 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-34896298
14.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Lancet Neurol
; 21(1): 42-52, 2022 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-34942136
15.
A Patient-Centered Evaluation of Meaningful Change on the 32-Item Motor Function Measure in Spinal Muscular Atrophy Using Qualitative and Quantitative Data.
Front Neurol
; 12: 770423, 2021.
Artículo
en Inglés
| MEDLINE | ID: mdl-35111124
16.
Outcome measures based on digital health technology sensor data: data- and patient-centric approaches.
NPJ Digit Med
; 3: 97, 2020.
Artículo
en Inglés
| MEDLINE | ID: mdl-32715091
17.
An overview of using qualitative techniques to explore and define estimates of clinically important change on clinical outcome assessments.
J Patient Rep Outcomes
; 3(1): 16, 2019 Mar 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-30830492
18.
Correction to: Twoyear efficacy and safety of risdiplam in patients with type 2 or nonambulant type 3 spinal muscular atrophy (SMA).
J Neurol
; 270(5): 2547-2549, 2023 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-37071150
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