Cargando…

Looking Forward to New Therapies: A Personal Perspective on the Translational Landscape for Muscular Dystrophies

Detalles Bibliográficos
Autor principal:	Bushby, Kate
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2015
Materias:	Perspective
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5240615/ https://www.ncbi.nlm.nih.gov/pubmed/27858762 http://dx.doi.org/10.3233/JND-150096

Ejemplares similares

Precision Medicine and Exercise Therapy in Duchenne Muscular Dystrophy
por: Kostek, Matthew
Publicado: (2019)

GVC transformation and a new investment landscape in the 2020s: Driving forces, directions, and a forward-looking research and policy agenda
por: Zhan, James X.
Publicado: (2021)

The importance of genetic diagnosis for Duchenne muscular dystrophy
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2016)

Mechanisms of muscle weakness in muscular dystrophy
por: Goldstein, Jeffery A., et al.
Publicado: (2010)

Note from the Editor: Looking Forward
por: Burkhart, James G.
Publicado: (2006)

Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy
por: Ramos-Platt, Leigh, et al.
Publicado: (2022)

Muscle-on-a-chip devices: a new era for in vitro modelling of muscular dystrophies
por: Fernández-Costa, Juan M., et al.
Publicado: (2023)

New aspects on patients affected by dysferlin deficient muscular dystrophy
por: Klinge, Lars, et al.
Publicado: (2009)

The Barancik lecture: Comorbidity in multiple sclerosis—Looking backward, looking forward
por: Marrie, Ruth Ann
Publicado: (2023)

Improving recognition of Duchenne muscular dystrophy: a retrospective case note review
por: van Ruiten, Henriette J A, et al.
Publicado: (2014)

Vision 2020: looking back and thinking forward on The Lancet Oncology Commissions
por: Gospodarowicz, Mary K, et al.
Publicado: (2020)

Quantifying the burden of caregiving in Duchenne muscular dystrophy
por: Landfeldt, Erik, et al.
Publicado: (2016)

Myeloid-Derived Suppressor Cells (MDSCs) in Ovarian Cancer—Looking Back and Forward
por: Okła, Karolina
Publicado: (2023)

Imperatives for DUCHENNE MD: a Simplified Guide to Comprehensive Care for Duchenne Muscular Dystrophy
por: Kinnett, Kathi, et al.
Publicado: (2015)

A closer look at cannabimimetic terpenes, polyphenols, and flavonoids: a promising road forward
por: Cavalli, Juliana, et al.
Publicado: (2020)

Compliance to Care Guidelines for Duchenne Muscular Dystrophy
por: Landfeldt, Erik, et al.
Publicado: (2015)

Reduced-intensity conditioning hematopoietic stem cell transplantation: looking forward to an international consensus
por: Chaudhry, Monazza, et al.
Publicado: (2015)

Adult care for Duchenne muscular dystrophy in the UK
por: Rodger, Sunil, et al.
Publicado: (2014)

Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders
por: Jablonka, Sibylle, et al.
Publicado: (2022)

The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle
por: Rybalka, Emma, et al.
Publicado: (2020)

LMNA-related muscular dystrophy: Identification of variants in alternative genes and personalized clinical translation
por: Cesar, Sergi, et al.
Publicado: (2023)

Reframing conservation physiology to be more inclusive, integrative, relevant and forward-looking: reflections and a horizon scan
por: Cooke, Steven J, et al.
Publicado: (2020)

Three decades of ethical, legal, and social implications research: Looking back to chart a path forward
por: Dolan, Deanne Dunbar, et al.
Publicado: (2022)

The burden of Duchenne muscular dystrophy: An international, cross-sectional study
por: Landfeldt, Erik, et al.
Publicado: (2014)

Intraventricular immunovirotherapy; a translational step forward
por: Bernstock, Joshua D., et al.
Publicado: (2023)

Attenuated muscle regeneration is a key factor in dysferlin-deficient muscular dystrophy
por: Chiu, Yen-Hui, et al.
Publicado: (2009)

Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis
por: Landfeldt, Erik, et al.
Publicado: (2016)

Evaluating a Stone of Hope: ICER’s 2019 Review of Treatments for Duchenne Muscular Dystrophy
por: Brandsema, John Frederick
Publicado: (2020)

Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy
por: Zaharieva, Irina T., et al.
Publicado: (2013)

Muscle-Derived Proteins as Serum Biomarkers for Monitoring Disease Progression in Three Forms of Muscular Dystrophy
por: Burch, Peter M., et al.
Publicado: (2015)

Prion-like domain disease-causing mutations and misregulation of alternative splicing relevance in limb-girdle muscular dystrophy (LGMD) 1G
por: Batlle, Cristina, et al.
Publicado: (2020)

Looking back — Looking forward — A new year
por: Homer, Caroline
Publicado: (2022)

The Central Dogma Decentralized: New Perspectives on RNA Function and Local Translation in Neurons
por: Holt, Christine E., et al.
Publicado: (2013)

Current Translational Research and Murine Models For Duchenne Muscular Dystrophy
por: Rodrigues, Merryl, et al.
Publicado: (2016)

Translating golden retriever muscular dystrophy microarray findings to novel biomarkers for cardiac/skeletal muscle function in Duchenne Muscular Dystrophy
por: Galindo, Cristi L., et al.
Publicado: (2015)

Muscular Dystrophy: Disease Mechanisms and Therapies
por: Pandey, Sachchida Nand, et al.
Publicado: (2015)

Advances in gene therapy for muscular dystrophies
por: Abdul-Razak, Hayder, et al.
Publicado: (2016)

Gene Therapy for Duchenne Muscular Dystrophy
por: Elangkovan, Nertiyan, et al.
Publicado: (2021)

Cardiac therapies for Duchenne muscular dystrophy
por: Shah, Md Nur Ahad, et al.
Publicado: (2023)

The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy: A Summary from the Institute for Clinical and Economic Review’s New England Comparative Effectiveness Public Advisory Council
por: Agboola, Foluso, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_853faabcvj9i3qkda05gotoc75