Investigational therapies for the treatment of spinal muscular atrophy

Expert Opinion on Investigational Drugs

Volumn 24, Issue 7, 2015, Pages 867-881

  Kaczmarek, Anna ^a   Schneider, Svenja ^a   Wirth, Brunhilde ^a   Riessland, Markus ^a  

^a UNIVERSITY OF COLOGNE   (Germany)

Author keywords

Aminoglycosides; Antisense oligonucleotides; BAY 55 9837; Bortezomib; Fasudil; Gabapentin; Histone deacetylase inhibitors; Hydroxyurea; Motor neuron; Olesoxime; Quinazolines; Riluzole; Salbutamol; Spinal muscular atrophy; Splicing; Suberoylanilide hydroxamic acid; Valproic acid; Viral gene therapy; Y 27632

Indexed keywords

4 (1 AMINOETHYL) N (4 PYRIDYL)CYCLOHEXANECARBOXAMIDE; AMINOGLYCOSIDE DERIVATIVE; ANTISENSE OLIGONUCLEOTIDE; BAY 559837; BORTEZOMIB; D 156844; FASUDIL; GABAPENTIN; HISTONE DEACETYLASE INHIBITOR; HUMAN GROWTH HORMONE; HYDROXYUREA; LDN 76070; MAMMALIAN TARGET OF RAPAMYCIN; OLESOXIME; PHOSPHATIDYLINOSITOL 3,4,5 TRISPHOSPHATE 3 PHOSPHATASE; PROTEASOME; PROTEIN; PROTEIN ROCK; QUERCETIN; QUINAZOLINE DERIVATIVE; RG 3039; RILUZOLE; SALBUTAMOL; SMN 1 C2; SMNC 1; SMNC 3; SURVIVAL MOTOR NEURON PROTEIN; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2; TC 007; UNCLASSIFIED DRUG;

CLINICAL TRIAL (TOPIC); DRUG EFFECT; DRUG RESEARCH; DRUG TARGETING; ENZYME INHIBITION; EXON; GENE; GENETIC TRANSCRIPTION; HUMAN; MUSCLE TRAINING; NONHUMAN; PATHOPHYSIOLOGY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHENOTYPE; PROTEIN EXPRESSION; REVIEW; RNA SPLICING; SMN1 GENE; SMN2 GENE; SPINAL MUSCULAR ATROPHY; VIRAL GENE THERAPY; ANIMAL; EXPERIMENTAL THERAPY; METABOLISM; MUSCULAR ATROPHY, SPINAL;

ANIMALS; HUMANS; MUSCULAR ATROPHY, SPINAL; SURVIVAL OF MOTOR NEURON 1 PROTEIN; THERAPIES, INVESTIGATIONAL;

EID: 84934964184     PISSN: 13543784     EISSN: 17447658     Source Type: Journal    
DOI: 10.1517/13543784.2015.1038341     Document Type: Review

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