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New Directions for SMA Therapy

New Directions for SMA Therapy

http://www.wikidata.org/entity/Q58779205

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New Directions for SMA Therapy

http://www.wikidata.org/entity/Q58779205

description

scientific article published on 31 August 2018

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wetenschappelijk artikel

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наукова стаття, опублікована в серпні 2018

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name

New Directions for SMA Therapy

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New Directions for SMA Therapy

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type

label

New Directions for SMA Therapy

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New Directions for SMA Therapy

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prefLabel

New Directions for SMA Therapy

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New Directions for SMA Therapy

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Journal of Clinical Medicine

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New Directions for SMA Therapy

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Sonia Messina

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

Identification and characterization of a spinal muscular atrophy-determining gene

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

Temporal requirement for high SMN expression in SMA mice

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial.

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study.

Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

The small-molecule fast skeletal troponin activator, CK-2127107, improves exercise tolerance in a rat model of heart failure.

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251

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scholarly article

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10.3390/JCM7090251

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9

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7

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2018-08-31T00:00:00Z

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30200278

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6162810

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