Rosie was the first of three HNRNPH2 patients to receive treatment with an n-Lorem ASO. Each of them, pioneering a path to treatment for others. Rosie’s parents, Nicole and Bobby, discuss their daughter’s journey and how they have adapted as a family to meet the needs of their daughter’s condition. Nicole is a pediatrician herself and admits even she doesn’t always have the answers to caring for a child with a nano-rare disease — a reality that resonates with many nano-rare families.

This episode is proudly sponsored by Hongene Biotech

• 2:00 Robert left his job to help care for Rosie full-time 
• 7:36 Nicole is a pediatrician and it’s difficult for her to not regularly attend Rosie’s appointments 
• 11:16 Rosie’s diagnosis of HNRNPH2 
• 16:00 Even as a pediatrician, Nicole finds nano-rare diseases complex and difficult to navigate 
• 19:29 What is HNRNPH2? 
• 26:40 Coming to terms with the diagnosis 
• 29:35 Weighing the potential risk/benefit and making the decision that Rosie will be the first HNRNPH2 patient treated with an experimental medicine 
• 37:05 How Rosie is doing after three ASO treatment doses 
• 40:35 Potential for a placebo effect and allowing multiple observers to notice changes 

Nicole Glenn, MD, is a pediatrician at Sutter Health with 14 years of experience. She earned her bachelor’s degree in Physiology at the University of Colorado, Boulder, completed medical school at Rush University in Chicago, and residency at Stanford University, Lucile Packard Children’s Hospital.

Robert “Bobby” Glenn studied Molecular and Cellular Biology at the University of Colorado, Boulder, but ultimately found his passion working in various roles in business development and marketing in the tech industry.

Their daughter was diagnosed with HNRNPH2 in 2019. They’ve watched their determined daughter work hard for every little achievement and are proud of her accomplishments. They are both involved with the Yellow Brick Road Project (HNRNPH2 Foundation) with a mission to fund research to identify, understand, treat those impacted by HNRNPH2 mutations. Bobby is the director of marketing & communications, and a board director. Nicole serves as an International and Western USA delegate.

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