SMA Europe provides funding for five new research projects in SMA
Awards come as part of organization's 12th Call for Research proposals
SMA Europe is funding five new research projects that are intended to advance understanding of spinal muscular atrophy (SMA) and guide the development of new therapies.
The funding comes as part of SMA Europe’s 12th Call for Research proposals, which the advocacy organization runs every other year to support SMA research. As in years past, applications are reviewed by a panel of scientific experts and the review process this year included a panel of patients and caregivers who have knowledge of the science of SMA and/or experience in advocacy.
“SMA Europe is thrilled to announce awardees of our 12th Call for Research. It is inspiring to see such extraordinary researchers pushing the boundaries of scientific discovery,” Nicole Gusset, CEO of SMA Europe, said in a press release to SMA News Today.
SMA is a neuromuscular disease marked by symptoms that include muscle weakness and wasting. Over the last decade or so, the landscape of SMA care has been radically transformed by the approval of a number of treatments that can slow the disease’s progression. Despite these advances, many challenges still face face the SMA community.
“Even with the treatments now available, fundamental research remains essential to our SMA community,” Gusset said. “Only by continuously supporting innovative approaches can we pave the way for breakthroughs that may one day further transform the landscape for all those living with SMA.”
Finding new treatments for SMA
SMA treatments can slow or even halt disease progression, but their effects can vary from person to person. One of the newly funded projects seeks to identify new biomarkers to track SMA progression and treatment responses. That project will be led by Gabriella Viero, PhD, at the National Research Council in Trento, Italy.
Another project will focus on how SMA affects muscle stem cells, which are specialized cells that can help repair damaged muscle tissue. That project, which will explore if available SMA treatments help preserve muscle stem cells, is led by Nathalie Didier, PhD, at the Institut national de la santé et de la recherche médicale (INSERM) in France.
At the cellular level, some of the earliest problems in SMA affect the neuromuscular junction, which is where nerve cells connect with muscle cells to control muscle movement. A project led by Morgan Gazzola, PhD, at the Institute for Stem cell Therapy and Exploration of Monogenic diseases in France, will use a new cell level to better understand how the neuromuscular junction is affected in SMA.
Simon Parson, PhD, at the University of Aberdeen in the U.K., will be leading a study that seeks to better understand how SMA affects blood vessels, with a particular focus on how these defects may cause or aggravate SMA symptoms.
At the molecular level, calcium plays a key role in muscle contractions. The fifth project will use a fish model of SMA, along with human cells, to test whether calcium-modulating drugs might be part of SMA treatment. That project will be led by Sorana Ciura, PhD, at Institut Imagine in France.
About the Author
