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URMC / Clinical & Translational Science Institute / Stories / March 2017 / URMC to Host Technology & Rare Neurological Diseases Symposium

URMC to Host Technology & Rare Neurological Diseases Symposium

Hand holding light bulb with illuminated brain insideThe Technology & Rare Neurological Diseases Symposium (TRNDS), taking place Friday, May 12, aims to spark a national discussion among patients, researchers, and policy experts on how to accelerate clinical trials for rare diseases that attack the nervous system. New technologies may ease the burden of traveling to research sites for patients and increase the flow of information about these diseases for researchers and healthcare providers.

Finding enough patients to conduct clinical studies on rare diseases can be very difficult. Patients are spread far and wide and are often required to travel long distances or even move to access care and participate in clinical trials. And patients with rare neurological diseases often have physical and/or mental disabilities that make travel even more burdensome.

These difficulties lead to a lack of “natural history” knowledge about these diseases – what they are, who they impact, how symptoms unfold over time.

“Natural history is a real challenge for rare diseases,” said Erika Augustine, M.D., M.S., symposium organizer and Child Neurologist at the URMC Center for Human Experimental Therapeutics. “Understanding natural history is very important for developing clinical trials, so you target the right symptoms, know how to measure those symptoms, and know that these are factors that actually matter to patients and families.” 

TRNDS will bring together patients, patient advocates, clinical research policy and regulatory experts, and researchers from academia, biotechnology, and pharmaceutical industries to brainstorm ways to apply novel mobile health technologies to rare neurological diseases. Putting these key players together will hopefully foster new collaborations and new ideas.

At the symposium, biotechnology and pharmaceutical industry leaders will discuss conducting virtual visits in site-less clinical trials, building digital platforms to engage with patients and the Electronic Health Record, and developing wearable technologies to measure signs of disease. These technological advances could reduce or eliminate travel burden on patients, increase our natural history knowledge about rare neurological diseases, and offer new solutions for tracking symptoms.

While these technologies have great potential to benefit patients and research participants, attention to privacy concerns is important. That is one reason it is crucial to have these discussions with all of the key players - including patients and policy experts.  

Symposium organizers hope this event will be a step toward increasing patient access to care and ability to participate in research that may lead to innovative therapies.

Technology and Rare Neurological Diseases Symposium (TRNDS)
Friday, May 12, 2017
7:30 am – 3:45 pm
Helen Wood Hall Auditorium, University of Rochester

TRNDS is supported in part by conference grants from BioMarin Pharmaceutical, Burroughs Wellcome Fund, and the UR CTSI along with other sponsors.

Michael Hazard | 3/29/2017

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