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perspective thérapeutiqueSynonyme(s)perspective thérapeutique ;piste thérapeutique therapeutic prospects |
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Modulators of calpain activity: inhibitors and activators as potential drugs
Dokus LE, Yousef M, Banoczi Z
Expert opinion on drug discovery, 2020
Correction of RNA-Binding Protein CUGBP1 and GSK3beta Signaling as Therapeutic Approach for Congenital and Adult Myotonic Dystrophy Type 1
Timchenko L
International Journal of molecular sciences, 2019, 21, 1
Spinal muscular atrophy with respiratory distress type 1: Clinical phenotypes, molecular pathogenesis and therapeutic insights
Saladini M, Nizzardo M, Govoni A, et al.
Journal of cellular and molecular medicine, 2019
Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells
Pagliarini V, Guerra M, Di Rosa V, et al.
Journal of neurochemistry, 2019
MicroRNA-Based Therapeutic Perspectives in Myotonic Dystrophy
Lopez Castel A, Overby SJ, Artero R
International Journal of molecular sciences, 2019, 20, 22
Muscular dystrophy: new treatments, new hopes
Collectif
Lancet (London, England), 2019, 394, 10213, p 1966
Molecular Approaches for the Treatment of Pompe Disease
Bellotti AS, Andreoli L, Ronchi D, et al.
Molecular neurobiology, 2019
Molecular Mechanisms and Therapeutics for SBMA/Kennedy's Disease
Arnold FJ, Merry DE
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2019
Increased Muscleblind levels by chloroquine treatment improve myotonic dystrophy type 1 phenotypes in in vitro and in vivo models
Bargiela A, Sabater-Arcis M, Espinosa-Espinosa J, et al.
Proceedings of the national Academy of sciences of the United States of America, 2019
Current and emerging therapies in Becker muscular dystrophy (BMD)
Angelini C, Marozzo R, Pegoraro V
Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 2019, 38, 3, p 172
Cardiac Pathophysiology and the Future of Cardiac Therapies in Duchenne Muscular Dystrophy
Meyers TA, Townsend D
International Journal of molecular sciences, 2019, 20, 17
Zoom sur... la recherche dans la maladie de Charcot-Marie-Tooth
Myoinfo (AFM-Téléthon), Vallat JM
Zoom sur ..., Savoir & Comprendre, 2019, 46
233rd ENMC International Workshop: Clinical Trial Readiness for Calpainopathies, Naarden, The Netherlands, 15-17 September 2017
Lostal W, Urtizberea JA, Richard I
Neuromuscular disorders : NMD, 2018
Selective modulation of the androgen receptor AF2 domain rescues degeneration in spinal bulbar muscular atrophy
Badders NM, Korff A, Miranda HC, et al.
Nature medicine, 2018
Ubiquitination of MBNL1 Is Required for Its Cytoplasmic Localization and Function in Promoting Neurite Outgrowth.
Wang PY, Chang KT, Lin YM, et al.
Cell reports, 2018, 22, 9, p. 2294-2306
Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy
Torres-Torronteras J, Cabrera-Pérez R, Vila-Julia F, et al.
Human gene therapy, 2018
Protein-anchoring therapy to target extracellular matrix proteins to their physiological destinations
Ito M, Ohno K
Matrix biology : journal of the International Society for Matrix Biology, 2018
Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice
Tasfaout H, Lionello VM, Kretz C, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2018
A Pilot Study of Tranilast for Cardiomyopathy of Muscular Dystrophy
Matsumura T, Matsui M, Iwata Y, et al.
Internal medicine (Tokyo, Japan), 2018, 57, 3, p 311
Small-molecule flunarizine increases SMN protein in nuclear Cajal bodies and motor function in a mouse model of spinal muscular atrophy.
Sapaly D, Dos Santos M, Delers P, et al.
Scientific Reports, 2018, 8, 1, p. 2075
Zidovudine ameliorates pathology in the mouse model of Duchenne muscular dystrophy via P2RX7 purinoceptor antagonism
Al-Khalidi R, Panicucci C, Cox P, et al.
Acta neuropathologica communications, 2018, 6, 1
X-Linked Spinal and Bulbar Muscular Atrophy: From Clinical Genetic Features and Molecular Pathology to Mechanisms Underlying Disease Toxicity
La Spada AR
Advances in experimental medicine and biology, 2018, 1049, p 103
