Cure SMA Publishes Booklet with Research Process Overview

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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Cure SMAAfter working for 30 years funding research projects dedicated to spinal muscular atrophy (SMA) and facing the challenges related to it, Cure SMA is launching a new booklet to assist in the introduction of the research process. Advancing Research Toward a Treatment and Cure is available online and offers a new perspective on the scientific advancements related to SMA.

The nonprofit organization is dedicated to financing research projects that are expected to improve treatment of SMA or find a cure for the disease. The booklet aims to provide new insights about the research process in order to accelerate, as well as summarize the latest therapeutic advancements, including the awarding of research grants and the initiation of clinical trials.

As part of the effort to create the booklet, Cure SMA conducted an assessment of new research projects presently being worked on, as well as the organization’s potential contributions to the global effort to cure the disease.

Even though SMA treatment options were limited when Cure SMA began its work with researchers, the organization has steadily contributed to accelerating the process of drug development with its funding grants. Despite the fact that the majority of investigational drugs are not successful in clinical trials and only about 10% of the drugs end up receiving the approval of the U.S. Food and Drug Administration (FDA), there are currently 17 drug treatments accepted for SMA, 15 of which have been approved over the last 15 years.

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Given the numerous challenges associated with the research process, Cure SMA believes that one of the steps toward better and faster results involves examining a greater number of compounds. In addition, another major obstacle is funding. Receiving financial support to conduct the studies needed is a challenge that several research projects face.

To combat this, Cure SMA works with a strategy based on volume, balance, and leverage in order to enable the promotion of a series of options and diverse approaches. In addition, the organization aims to present the advantages of supporting SMA, making it an attractive investment for companies. Despite the obstacles, the organization believes that their strategic investments are resulting in tangible results through the increase of therapy pipelines.

Cure SMA has made the booklet available online as a PDF, as well as printed if requested through [email protected]. The organization will continue its work of supporting both patients and families who suffer with the disease, while providing grants to researchers who are focused on finding a cure for the disease.

The organization has recently been awarded a $2,500 grant from the oil and gas company Newfield Exploration, to be invested in Cure SMA’s main mission. The grant was awarded through the Newfield Foundation, an organization formed by Woodlands, Texas-based Newfield Exploration in 2001, with the purpose of improving the quality of life in communities through key donations.