Ask A Doc: May 3, 2021 at 5pm ET. 

Facebook Live Event

(Must be a member to attend)

https://www.facebook.com/groups/BDSRA

 

David A Pearce, PhD

President of Innovation, Research, & World Clinic

 

David.Pearce@sanfordhealth.org

 

 

David Pearce is President of Innovation, Research, & World Clinic for Sanford Health.  He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986.  He gained his PhD in 1990 at the University of Bath, UK, and did postdoctoral training at the University of Rochester, U.S., and Oxford University, UK.

Dr. Pearce heads a leading lab in Batten disease research.  He has been researching Neuronal Ceroid Lipofuscinosis (Batten disease) since 1997.  His research has led to the first clinical trial for Juvenile Batten disease.  He has published over 100 research papers on Batten disease. He also oversees a national registry for rare diseases known as the Coordination of Rare Diseases at Sanford (CoRDS).   He has served on numerous NIH review committees, has organized rare disease workshops for the National Institute for Neurological Disorders and Stroke (NINDS) arm of the National Institutes of Health (NIH) and is currently the vice chair of the consortium assembly for the International Rare Diseases Research Consortium (IRDiRC). 

 

As President of Innovation, Research, & World Clinic at Sanford, he oversees the development of research programs, including more than 450 researchers, eight research centers and more than 300 ongoing clinical trials. 

 

A friendly reminder to join us on May 10 from 10am – 2pm ET for a workshop on the Patient Advocates’ Role in Advancing Gene Therapy taking place before the ASGCT Annual Meeting. We are excited to offer two discount options on the registration fee:

Waived Registration Fee for individuals with a rare disease, caregivers of a person with a rare disease, and volunteer advocates. Learn more!

Reduced Rate for Patient Advocates by selecting that you are patient advocate when registering you can attend for only $25.

Natural History Study enrolling virtually now, until it’s safe for families to travel.

https://clinicaltrials.gov/ct2/show/NCT03822650?cond=cln5&draw=2&rank=1

 

Caregiver Focus Groups for CLN5

Dates will be determined by the families’ availability. View article here.

 

Neurogene is currently working with RTI Health Solutions (RTI‑HS), an independent, nonprofit research organization to better understand the disease progression of Batten disease subtype CLN5 from the caregiver’s perspective.

Caregivers of children (either currently living or deceased within the past 12 months) with Batten disease subtype CLN5 who are eligible for this study (Qualitative Interviews With Caregivers of Children With CLN5-Specific Batten Disease) and live in the United States will be invited to participate in a telephone interview with two RTI‑HS interviewers lasting approximately 90 minutes. Interview participants will receive an electronic gift card (e.g., Visa, Amazon, or other online store of choice) for $150 in appreciation for their time.

During the interviews with RTI‑HS, caregivers will be asked to describe their child’s and their own experiences surrounding the journey to and following diagnosis, including observed signs and symptoms of the disease, delays in any of the developmental milestones, and perceived impacts of Batten disease subtype CLN5 on the child, caregiver, and family.

The discussion will be audio recorded and transcribed to ensure the interviewers capture the important points that participants share and to help write a report summarizing the results of the interviews. Participants’ names and any other identifying information will not be associated with the transcripts. The audio recordings will be destroyed once a final report is written.

 

If you are interested in participating in a telephone interview, please call Danielle at L&E Research at 720-647-3228 who will ask you a few questions to confirm that you qualify for this study. If you qualify, L&E Research will schedule you for an interview at a day and time that is convenient for you.

Taysha Gene Therapies is grateful for the caregivers who have shared their CLN1 disease experiences with our team. In doing so, they helped to advance Taysha’s understanding of a CLN1 disease diagnosis, symptoms, and management. Such insights gleaned from caregivers are important to consider when selecting endpoints and designing a clinical trial for a novel intervention in a disease where there are no available treatments.  Pending the results of its meetings with regulators, Taysha hopes to initiate a Phase 1/2 clinical trial for TSHA-118 in CLN1 disease in the second half of 2021.  Patients & their families are the core of Taysha.  This year, on Rare Disease Day, we announced #RareAlly as our way of recognizing individuals and groups who inspire us daily.  Visit #RareAlly to learn more about Taysha’s inspiration.