Kaczmarek, Anna, Schneider, Svenja, Wirth, Brunhilde ORCID: 0000-0003-4051-5191 and Riessland, Markus ORCID: 0000-0003-2592-5045 (2015). Investigational therapies for the treatment of spinal muscular atrophy. Expert Opin. Investig. Drugs, 24 (7). S. 867 - 882. ABINGDON: TAYLOR & FRANCIS LTD. ISSN 1744-7658

Full text not available from this repository.

Abstract

Introduction: Currently, there is no cure available for the hereditary neurodegenerative disease proximal spinal muscular atrophy (SMA), which is the number one genetic killer in early childhood. However, growing knowledge of SMA pathophysiology has opened new avenues for potential therapeutic interventions. Areas covered: This review summarizes a variety of investigational therapeutic approaches for SMA. Focusing on the current state-of-the-art applications, the authors discuss the outcome of the first clinical interventions and compare the first results from the newest strategies. The achievements of the investigational drugs highlighted in this article were deduced from original articles, pharmaceutical company press releases and clinical trial results. Expert opinion: Nearly two decades after the discovery of the disease causing gene survival motor neuron 1, many therapeutic options for SMA have been developed, some of which made it to clinical trials but could not prove their promising experimental results. Recently, big research efforts from academia, government and the pharmaceutical industry have led to the development of highly promising compounds that are currently in clinical trials, and which could lead to feasible treatment options in the future.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Kaczmarek, AnnaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schneider, SvenjaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wirth, BrunhildeUNSPECIFIEDorcid.org/0000-0003-4051-5191UNSPECIFIED
Riessland, MarkusUNSPECIFIEDorcid.org/0000-0003-2592-5045UNSPECIFIED
URN: urn:nbn:de:hbz:38-399628
DOI: 10.1517/13543784.2015.1038341
Journal or Publication Title: Expert Opin. Investig. Drugs
Volume: 24
Number: 7
Page Range: S. 867 - 882
Date: 2015
Publisher: TAYLOR & FRANCIS LTD
Place of Publication: ABINGDON
ISSN: 1744-7658
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
SURVIVAL-MOTOR-NEURON; INCREASE SMN LEVELS; EXONIC SPLICING ENHANCER; MESSENGER-RNA; MOUSE MODEL; ANTISENSE OLIGONUCLEOTIDES; DISEASE SEVERITY; PROTEIN-LEVELS; ANIMAL-MODEL; THERAPEUTIC TARGETMultiple languages
Pharmacology & PharmacyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/39962

Downloads

Downloads per month over past year

Altmetric

Export

Actions (login required)

View Item View Item