Spinal muscular atrophy (SMA) is one of the rare neuromuscular disorder that affects certain parts of the nervous system, which controls the muscle movement. In the last few years, there has been an increase in the number of SMA cases across the globe. Spinal muscular atrophy is considered as one of the prominent genetic disorders resulting in mortality among infants. 

The spinal muscular atrophy market has been classified on the basis of symptoms into type 1, type 2, type 3, and type 4 spinal muscular atrophy. Type 1 SMA is referred as the most severe form that affects infants aged 1 to 6 months. As per the report, around 60% of the overall SMA cases are of type 1, in which infants do not live more than two years. Type 2 SMA is considered moderate, which affects children between 6 and 18 months. Type 3 and type 4 patients mostly have a normal life expectancy.

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The research study further discusses the pipeline of the spinal muscular atrophy. Early stage candidates and late stage candidates are the two major segments studied in the report. At present, ISIS-SMNRx by Ionis Pharmaceuticals, Inc. is the only late stage drug candidate present in the spinal muscular atrophy pipeline. Furthermore, three drugs are in phase 2 and three others are in phase 1 in the SMA pipeline. The rising research activities are expected to introduce more drug candidates in the pipeline, driving the overall market in the near future.

The research study gathers information from governments and corporate sources, including the U.S. and Europe clinical trial registries, Centers for Disease Control and Prevention (CDC), company press releases, World Health Organization (WHO), Families of SMA, CureSMA, PubMed, annual reports, and investor presentations. The analysis in the report offers inputs given by industry experts with their expertise in rare disease drug discovery.

In the last few years, Europe and the U.S. have witnessed significant increases in the number of diagnosed cases of spinal muscular atrophy. Thus, private organizations and governments are making efforts to create an awareness regarding the disease among the population. Furthermore, the rising confirmed carrier population in these regions are further anticipated to contribution substantially towards the growth of the spinal muscular atrophy market. In addition, the government incentives offered for developing therapeutic alternatives are projected to drive the overall market in the next few years.

Furthermore, the research study offers a detailed analysis of the players operating in the spinal muscular atrophy market. Some of the leading players are Ionis Pharmaceuticals, Inc., F. Hoffmann-La Roche Ltd., AveXis, Inc., Novartis AG, and Cytokinetics, Inc. The contact information, business strategies, product portfolio, financial status, SWOT analysis, and recent developments have been discussed in the research report. In addition, the potential candidates in clinic trials and pipeline for treatment of spinal muscular atrophy have also been covered in the research report.

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