“We’re rare disease families trying to do our best for every patient that gets submitted.”

Jeff Carroll, Ph.D., inherited a gene that will eventually lead to symptoms of Huntington’s Disease. Alongside researching this debilitating disease as an Associate Professor of Neurology at the University of Washington, he’s a Scientific Advisor for n-Lorem and member of the Access to Treatment Committee (ATTC) that helps screen and assess submitted patient applications.

• 2:45 Joining the Amy on a whim  
• 4:30 Serving in Kosovo and Germany
• 6:00 Learning that his mother was diagnosed with Huntington’s disease (HD)
• 10:25 Seeking information and diving into the world of Biology and HD
• 14:52 Deciding to have children when there was a chance that they’d inherit the disease and utilizing preimplantation genetic diagnosis (PGD)
• 18:30 Watching Ionis make initial progress on an ASO for Huntington’s disease
• 23:10 How Jeff became involved with n-Lorem
• 27:30 Most important things Jeff has learned during his role at n-Lorem
• 30:38 Helping people is motivating
• 32:11 Nano-rare patients teach us a lot about science
• 33:57 Jeff expects to receive an ASO treatment one day
• 35:22 n-Lorem is on your side 

Dr. Jeff Carroll is a scientific advisor for n-Lorem. Jeff began assisting n-Lorem’s access to treatment committee at a very early stage, given his passion for helping families with rare genetic diseases. This passion emerges from a personal connection – Jeff’s mother died after suffering from Huntington’s Disease, placing her 6 children at 50% risk of developing the disease. In 2003, Jeff learned that he inherited this mutation from his mother, sparking a passion to help understand the disease, and to help hasten the development of novel treatments for all HD patients.

Jeff is now an associate professor in the Department of Neurology at the University of Washington. Jeff’s primary scientific focus is on conducting translational studies in support of “Huntingtin Lowering” approaches to Huntington’s Disease, primarily through the use of antisense oligonucleotides. Jeff’s Ph.D. and postdoctoral studies all involved using mouse models to understand basic mechanisms of HD and preclinical testing of therapeutic interventions in these models. During his Ph.D., he trained with Michael Hayden (UBC) and his postdoctoral studies were conducted under the supervision of Marcy MacDonald (MGH, Harvard Medical School).

We cannot do
this alone

We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.