I recently had the honor of interviewing bodybuilder L.A. Aguayo over Zoom. He is 34 years old, has severe hemophilia B, and recently took second place in an international bodybuilding competition, the Arnold Amateur. I highly recommend his inspirational autobiography, “Finding Purpose in the Pain.” In his book,…

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

My youngest son, Caeleb, is 16 and preparing to begin his junior year in high school. School has been difficult for him since before kindergarten. With complications from hemophilia and an inhibitor, he missed numerous days of school through his third-grade year, which caused him to…

Venture philanthropy fund Pathway to Cures (P2C)) has invested $500,000 in Five Liters, which is developing noninvasive, nonpharmacological neuromodulation therapies for treating inheritable blood and bleeding disorders, such as hemophilia and von Willebrand disease (VWD). “Investing in Five Liters’ early-stage research is a critical step in addressing unmet…

I’m training to become a chaplain. I want to work in a hospital setting one day, which will allow me to serve patients facing medical challenges. I’ll also have the privilege of walking with those who are in their last days. Sitting with people who are struggling at the hands…

Last year, at the Hemophilia Federation of America‘s Mild Matters Summit in Oklahoma, I attended a session led by physical therapist Nancy Durben that left a lasting impact. Durben guided a group on a leisurely walk, urging us to savor the experience — to relish the sound…

People are suffering. The country is divided. States are imposing limits on items that may be purchased, and businesses are closed. If there were ever a year to skip Thanksgiving, this would be it. At least, that is what some people think, but I am not one of them. It…

CSL Behring has closed an agreement giving it global commercialization and licensing rights to AMT-061, an experimental gene therapy for hemophilia B now being tested in a Phase 3 clinical trial. The therapy’s developer, uniQure, will receive an upfront cash payment of $450 million from CSL…

Sobi and Sanofi’s philanthropic organization, Foundation S, have announced an additional donation of up to 100 million international units (IUs) of clotting factor treatment to support the World Federation of Hemophilia (WFH) Humanitarian Aid Program. This is the second renewal of the 10-year partnership that will continue…

A new mom recently called me to share her concerns and to ask questions about choosing a new treatment for her son. She needed a listening ear, and just thinking about the possibility that her little boy might never need an implanted port gave me chills. I thought…