The requested article has expired, and is no longer available. Any related articles, and user comments are shown below.
© Copyright 2022 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed without permission.Gene therapy for hemophilia gets FDA approval
By MATTHEW PERRONE WASHINGTON©2024 GPlusMedia Inc.
2 Comments
Login to comment
painkiller
Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding.
A new drug emicizumab does not have clotting factors but can help people with hemophilia A.
virusrex
If this becomes a reality the reduced costs are only part of the benefits, treatments for the patients all come with risks and inconveniences, so lots of patients have great expectations of gene therapy for their quality of life.
Spinal muscular atrophy, sickle cell anemia, hemophilia and some kinds of cancer, we are in a time where gene therapies are making diseases that have plagued humanity something that can be done with one treatment that lasts for years or even all life.