Emerging therapies and challenges in spinal muscular atrophy - Wikidata - wikimedia - TriplyDB

Emerging therapies and challenges in spinal muscular atrophy

Emerging therapies and challenges in spinal muscular atrophy

http://www.wikidata.org/entity/Q33578135

about

Spinal Muscular Atrophy: From Defective Chaperoning of snRNP Assembly to Neuromuscular Dysfunction.Inherited Paediatric Motor Neuron Disorders: Beyond Spinal Muscular Atrophy.Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review.Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD).Nusinersen: First Global Approval.Cure SMA and our patient community celebrate the first approved drug for SMA.Developing multidisciplinary clinics for neuromuscular care and research.Treatment Advances in Spinal Muscular Atrophy.Genomic disorders 20 years on-mechanisms for clinical manifestations.Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches.In Vivo Translatome Profiling in Spinal Muscular Atrophy Reveals a Role for SMN Protein in Ribosome Biology.MBNL expression in autoregulatory feedback loops.Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy.mRNP assembly, axonal transport, and local translation in neurodegenerative diseases.Nusinersen for the treatment of spinal muscular atrophy.Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular Atrophy.Recent Advances in Antisense Oligonucleotide Therapy in Genetic Neuromuscular Diseases.Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening.Financial, opportunity and psychosocial costs of spinal muscular atrophy: an exploratory qualitative analysis of Australian carer perspectives.

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P2860

Emerging therapies and challenges in spinal muscular atrophy

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2016 nî lūn-bûn

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2016年論文

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2016年論文

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2016年論文

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2016年論文

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2016年论文

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2016年论文

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Emerging therapies and challenges in spinal muscular atrophy

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Emerging therapies and challenges in spinal muscular atrophy

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Emerging therapies and challenges in spinal muscular atrophy.

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Emerging therapies and challenges in spinal muscular atrophy

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Emerging therapies and challenges in spinal muscular atrophy

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Emerging therapies and challenges in spinal muscular atrophy.

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Emerging therapies and challenges in spinal muscular atrophy

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Emerging therapies and challenges in spinal muscular atrophy

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Emerging therapies and challenges in spinal muscular atrophy.

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Annals of Neurology

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Emerging therapies and challenges in spinal muscular atrophy

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Kathryn J Swoboda

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Michelle A Farrar

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Susanna B Park

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Creative Commons Attribution-NonCommercial

P2860

Drug treatment for spinal muscular atrophy types II and III

Drug treatment for spinal muscular atrophy type I

The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins

A positive modifier of spinal muscular atrophy in the SMN2 gene

Oxidative Stress Triggers Body-Wide Skipping of Multiple Exons of the Spinal Muscular Atrophy Gene

A large animal model of spinal muscular atrophy and correction of phenotype.

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.

Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens

Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy

Cardiac defects contribute to the pathology of spinal muscular atrophy models

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355-368

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scholarly article

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10.1002/ANA.24864

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3

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81

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Thomas H. Gillingwater

Bradley J Turner

Matthew C Kiernan

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2016-12-27T00:00:00Z

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2017-03-01T00:00:00Z

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28026041

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spinal muscular atrophy

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5396275

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