
Ben Samelson-Jones, MD, PhD
1.1K posts

Ben Samelson-Jones, MD, PhD
@DrSamelsonJones
Pediatric Hematologist | Gene Therapist | Biochemist | Husband | 👩👦👧👶 🐕 Dad | #WilliamsSyndrome Advocate | Views my own










@DrDanMO Here are the survival curves presented at #ash24, which appear to separate within a day of IV FE. To me this seems early for a plausible biological effect and raise the concern that the cohorts are not sufficiently matched and thus confounded by indication



15y ago, a family in Padua was found to have a FIX variant (R338L) with 5-10x gain-of-function activity relative to WT. Now, in a ph2b trial of 54 pts w hemophilia B (FIX deficiency), single gene therapy dose of AAV5-delivered FIX R338L led to ↓63% in annualized bleeding nejm.org/doi/full/10.10… @NEJM #ASH25 (original family description nejm.org/doi/full/10.10…)




ASH Plenary - IV Iron safe in infections! meetings-api.hematology.org/api/abstract/v…

We lost someone extraordinary Ethan Neumann, a PhD candidate in my lab, worked tirelessly to study #fibrolamellar carcinoma, the cancer that would ultimately claim his life On the day before he died he officially became Dr. Neumann: myarchoan.wixsite.com/yarchoanlabs/a…


open.substack.com/pub/pauloffit/… The risks of discouraging the birth dose for hepatitis B vaccine.






“I had hemophilia for nearly five decades. I went to Philadelphia and had a 45-minute infusion, and my hemophilia was gone.” Genes-> Medicine @NatureMedicine nature.com/articles/s4159…

From discovery to safety, innovation takes center stage at the #WFHGlobalForum. 🧬 Experts @DrSamelsonJones & WFH VP, Medical, @gfp55, MD, PhD, unpack the promise of #GeneTherapy—and the global challenges of ensuring safe, equitable access. #BleedingDisorders
