How do cells communicate with each other? With trillions of cells in the human body, seamless collaboration is essential within this intricate cellular society. Cells work together to form organs, and when one organ needs another to perform a task, they send signals across distances—much like picking up the phone and calling someone. Organ Hotline

📞 Eyes: Hey, Brain? This is Eyes. We’re reading this post about how organs communicate, and it’s really interesting. 📞 Brain: Oh, show me more! What’s it say? 📞 Eyes: Apparently, organs send signals to each other to get stuff done. Like, the liver calls the stomach when it’s time to detox! 📞 Brain: Whoa, I had no idea they were so organized! Keep reading, I need all the details! 📞 Eyes: I’m on it! But it says they made a whole podcast episode on in. 📞 Brain: Alright, I’m sold! I'm signaling to press play. On This Episode We Discuss:

• How cells talk to each other
• Multicellular organisms
• Why cells create organs
• Organ to organ communication - Electrochemical
• Chemical signals - The endocrine system
• Hormones created by other organs

This episode is brought to you by Hongene Biotech: https://www.hongene.com/

Support n-Lorem and nano-rare patient: https://www.nlorem.org/donate/

Rosie was the first of three HNRNPH2 patients to receive treatment with an n-Lorem ASO, helping to pave the way for others. Her parents, Nicole and Bobby, share their daughter's journey and how they have adapted as a family to meet her unique needs. As a pediatrician, Nicole understands the complexities of medical care, yet she acknowledges that even she doesn’t always have the answers when it comes to raising a child with a nano-rare disease—a reality that resonates with many nano-rare families. On this episode we discuss:

2:00 Bobby 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

Donate to n-Lorem: https://www.nlorem.org/donate/ This episode is brought to you by Hongene Biotech: https://www.hongene.com/

Sloane was born with a KIF1A mutation and is a special case at n-Lorem—she was the first patient to receive a medicine originally designed for someone else. Her mother, Megan, and grandfather, Tom, join the Patient Empowerment Program to share her journey and provide an update on how she’s doing six months after treatment.

On This Episode We Discuss:

• Sloane’s KIF1A diagnosis
• Nano-rare diseases affect the entire family
• Sloane was able to use the same ASO as another KIF1A patient
• Finding n-Lorem through KIF1A.ORG
• Shocked that n-Lorem was able to create personalized treatments efficiently at cost
• Sloane’s case is unique in a variety of ways and the decision to treat
• How Sloane has responded to treatment
• Are better measurements of benefit needed for nano-rare patients?
• How Sloane has brought empathy, joy, and positivity to her family
• Tom unretired to help fund research and support
• The origins of ASO medicine is a long haul

Thank you to Hongene Biotech for sponsoring this patient story episode!

Make Hope Possible for nano-rare patients with a donation to n-Lorem.