BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…
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The gene therapies AMT-060 and AMT-061 both restored coagulation factor IX levels in a primate model of hemophilia B, but AMT-061 resulted in greater coagulation activity at the same dose, a new study reports. The study, “Enhanced Factor IX Activity Following Administration of AAV5-R338L “Padua” Factor IX…
https://www.youtube.com/watch?v=dpUNgYbsQms If you have a chronic illness, you know just how much energy it takes to complete your daily chores and common household tasks. In this video from newlifeoutlook, Jennifer shares some practical tips to help make life a little easier for anyone suffering from a chronic illness. MORE: What is…
Healthcare costs are comparable for male patients with hemophilia A who are treated with either Adynovate (rurioctocog alfa pegol) or Eloctate (efmoroctocog alfa), according to a real-world study in the U.S. “Results from this analysis may be used to inform product coverage and treatment decisions valuable for both…
Hemophilia in children is diagnosed at an average age of nine months, and most children are diagnosed by their second birthday. If your pediatrician suspects your child has hemophilia, you may be concerned about their first appointment with the hematologist. To ease your worries, we’ve put together some tips to help you through…
Some moments in my life are ingrained in my soul, such as the births of my sons, my wedding day, the mornings when each of my parents died, and when I heard doctors utter the word “hemophilia.” These are points along my journey that changed the trajectory of my life…
I am a work-from-home mom, which means that I have two full-time positions as a “mom” and a “worker.” I am also a full-time carer to my husband with hemophilia. Thankfully, that position affords me several “leave credits” as my husband is not always injured. (Hopefully, the investment we’ve made…
Grifols is expanding its commitment to the World Federation of Hemophilia (WFH) Humanitarian Aid Program, donating at least 240 million international units (IU) of blood clotting factors over eight years to consistently treat some 3,000 bleeding disorder patients in developing countries. The contribution, starting in 2022 and running through…
A recent news story caught my attention. The report highlighted local athletes who unfortunately sustained injuries while serving in the military. These amazing women and men play basketball, tennis, and several other sports while in wheelchairs. Watching these phenomenal veterans play with determination and the physical prowess of professional athletes…
Good thing this column isn’t a podcast because I’m not speaking today. Last night, I chomped down on my tongue for the second time in two months. Not a big deal, you might say. True. Unless you have a bleeding disorder like hemophilia B, von Willebrand disease, or,…