Lysosomal Disease Network Awarded $5.8 Million

RDR Staff

University of Minnesota’s Lysosomal Disease Network announced it will continue to receive funding from the U.S. National Institutes of Health (NIH)’s Rare Diseases Clinical Research Network. The funding will total $5.8 million over the next 5 years.
The Lysosomal Disease Network is part of the University of Minnesota Medical School’s Department of Pediatrics. Principal investigator Chester B. Whitley, PhD, MD said:

“The renewed NIH funding will permit our research and educational efforts to grow, enhancing progress towards effective treatment and, ultimately, cures for these devastating diseases.”

Over the next 5 years, the Lysosomal Disease Network researchers will conduct 14 clinical research studies. Six of the studies will be led by clinical researchers at the University of Minnesota.

About the Lysosomal Disease Network

The Lysosomal Disease Network was founded in Minneapolis in 2004 to create synergies and increase efficiency among  U.S.-based lysosomal disease research efforts while also training the next generation of lysosomal disease physicians and researchers. The Network, while based in Minnesota, relies heavily on its collaborations and synergies with other institutes and organizations. The rarity of each lysosomal disease means that no single medical center will to see sufficient numbers of patients for a given disease making it necessary to have a network. During the next 5 years, the Lysosomal Disease Network is coordinating and supporting 13 leading researchers and their teams, located at 8 major medical research institutions across the United States, and at numerous participating other sites.
The Lysosomal Disease Network also works closely with patient advocacy groups.
Barbara Wedehase, executive director of the National MPS Society said:

 "Funding from the NIH to the LDN has resulted in innovative findings in lysosomal diseases which will ultimately improve the lives of those affected through new treatments and improved medical care; we look forward to continued progress and successes over the next five years."

Margie Frazier, the executive director of the Batten Disease Support and Research Association (BDSA), said:

 “The LDN fosters collaborations between researchers, the NIH, industry and patient and family advocacy groups — collaborations that are essential for driving new discoveries. Having strong voices from the patient community informs the long-term success of treatments and cures for the lysosomal diseases.”

Mark Dant, volunteer executive director of The Ryan Foundation, said:

 “NIH funding for the LDN over the years has improved the quality of life for many in the Lysosomal Storage Disease community. The news of the NIH continued support will undoubtedly move us all closer to the ultimate goal, which is to treat and cure each of these diseases in our lifetime.”

 About Lysosomal Diseases

Lysosomal diseases are a collection of more than 40 genetic diseases with incidence rates ranging from 1 in 20,000 (Gaucher disease) to 1 in 300,000 (Wolman disease) live births. Lysosomes are organelles responsible for the physiologic turnover of cell constituents (i.e., the garbage trucks of the cell). Patients deficient in a specific enzyme needed in the lysosomes often have a buildup of one compound in the cell. Since these organelles require a multitude of enzymes to function, there are numerous lysosomal diseases that are often defined by the specific enzyme that is lacking.
Some of the Lysosomal Diseases that the network studies include:
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