TSC Roundtable Discussion: Transforming the Future of TSC Care, Research

Tuberous sclerosis complex (TSC), a genetic disorder that causes tumors to form in different organs, affects nearly 1 million people worldwide and approximately 50,000 in the United States alone. Founded in 1974, the TSC Alliance has been a leader in the rare disease community and a comprehensive resource for individuals and caregivers affected by TSC.

The Alliance has a proven track record of accelerating research, strengthening care, and empowering the community. Since 1984, it has funded more than $27.7 million in TSC research and has also leveraged $446 million in additional federal funding through the National Institutes of Health and the Department of Defense Congressionally Directed Medical Research Program. Other notable achievements include establishing the first TSC Natural History Database, the first clinical network for patient care, clinical studies and clinical trials, and sponsoring research that helped identify the 2 genes that cause TSC.

As part of continuing to help transform the lives of those affected by TSC, the organization has committed to investing $40 million over 7 years to accelerate the discovery and development of improved therapies for TSC. The TSC Alliance’s campaign priorities are divided into 4 sections, including natural history database and biosample repository, clinical research, the Preclinical Consortium, and innovative research.

To spread the word about the several ongoing campaigns and advancements within the field, NeurologyLive partnered with the TSC Alliance for a roundtable discussion that featured experts Peter Crino, MD, and Steven Roberds, PhD. Crino, chairman of the Department of Neurology at the University of Maryland, and Roberds, chief executive officer of the TSC Alliance, have both been a part of the field for at least 2 decades. The duo sat down to converse about several topics within the space, including:

• Current knowledge of the disease
• Diagnosis of TSC and challenges associated with this process
• TSC Alliance’s Strategic Research Plan
• Importance of the Preclinical Consortium
• An assessment of the state of research
• Changing disease course through prenatal diagnosis
• The clinical pipeline of investigational TSC agents
• The future of clinical care for TSC

To learn more about how the efforts of the TSC Alliance are helping shape the future, click here.