Detalhe da pesquisa
1.
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.
Lancet
; 399(10329): 1049-1058, 2022 03 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-35279258
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.
Artigo
em Inglês
| MEDLINE | ID: mdl-34311756
3.
Delays in diagnosis of Duchenne muscular dystrophy: An evaluation of genotypic and sociodemographic factors.
Muscle Nerve
; 61(1): 36-43, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31573675
4.
Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy.
Proc Natl Acad Sci U S A
; 112(23): 7153-8, 2015 Jun 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-26039989
5.
Gene therapy companies have an ethical obligation to develop expanded access policies.
Mol Ther
; 29(4): 1367-1369, 2021 04 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33714373
6.
Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.
J Neuromuscul Dis
; 11(2): 499-523, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38363616
7.
Improving the Translational Medicine Process: Moving Patients From "End-Users" to "Engaged Collaborators".
Front Med (Lausanne)
; 6: 110, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-31165071
8.
Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022.
J Neuromuscul Dis
; 11(1): 233-252, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-37980681
9.
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy.
Neurology
; 89(17): 1811-1820, 2017 Oct 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-28972192
10.
Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy.
PLoS Curr
; 92017 Jan 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-28228973
11.
Improving Access and Quality of Health Care in the United States: Shared Goals Among Patient Advocates.
Patient
; 14(5): 687-690, 2021 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33083996
12.
Mothers' psychological adaptation to Duchenne/Becker muscular dystrophy.
Eur J Hum Genet
; 24(5): 633-7, 2016 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-26306645
13.
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
Lancet Neurol
; 15(8): 882-890, 2016 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-27302365
14.
Imperatives for DUCHENNE MD: a Simplified Guide to Comprehensive Care for Duchenne Muscular Dystrophy.
PLoS Curr
; 72015 Aug 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-26331093
15.
Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints.
Orphanet J Rare Dis
; 10: 16, 2015 Feb 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-25757705
16.
How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.
Orphanet J Rare Dis
; 10: 82, 2015 Jun 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-26104810
17.
What compassionate use means for gene therapies.
Nat Biotechnol
; 37(4): 352-355, 2019 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-30940936
18.
1st Workshop on Upper-Extremity Assistive Technology for People with Duchenne: State of the art, emerging avenues, and challenges: April 27th 2015, London, United Kingdom.
Neuromuscul Disord
; 26(6): 386-93, 2016 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-27133663
19.
Quality assurance for Duchenne and Becker muscular dystrophy genetic testing: development of a genomic DNA reference material panel.
J Mol Diagn
; 13(2): 167-74, 2011 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-21354051
20.
Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy.
Neuromuscul Disord
; 25(1): 96-105, 2015 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-25307856