Moving towards treatments for spinal muscular atrophy: Hopes and limits

Expert Opinion on Emerging Drugs

Volumn 20, Issue 3, 2015, Pages 353-356

  Wirth, Brunhilde ^a   Barkats, Martine ^b   Martinat, Cecile ^c   Sendtner, Michael ^d   Gillingwater, Thomas H ^e  

^a UNIVERSITY OF COLOGNE   (Germany)

^b SORBONNE UNIVERSITÉ   (France)

^c INSERM   (France)

^d UNIVERSITY HOSPITAL WÜRZBURG   (Germany)

^e UNIVERSITY OF EDINBURGH   (United Kingdom)

Author keywords

antisense oligonucleotide therapy; gene modifier; gene therapy; neonatal screening; neuromuscular disorder; pharmacotherapy; SMN1; SMN2; spinal muscular atrophy

Indexed keywords

HISTONE DEACETYLASE INHIBITOR; OLESOXIME; SURVIVAL MOTOR NEURON PROTEIN; SMN1 PROTEIN, HUMAN; SMN2 PROTEIN, HUMAN; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2;

EDITORIAL; GENE REPLACEMENT THERAPY; HUMAN; MOLECULARLY TARGETED THERAPY; MOTONEURON; NEWBORN SCREENING; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PROTEIN BLOOD LEVEL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SPINAL MUSCULAR ATROPHY; SYSTEMIC DISEASE; ANIMAL; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; MUSCULAR ATROPHY, SPINAL; NEWBORN; PATHOPHYSIOLOGY; PROCEDURES;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; HUMANS; INFANT, NEWBORN; MUSCULAR ATROPHY, SPINAL; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 84940388753     PISSN: 14728214     EISSN: 17447623     Source Type: Journal    
DOI: 10.1517/14728214.2015.1041375     Document Type: Editorial

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