Use of real‐world evidence in regulatory decisions for rare diseases in the United States—Current status and future directions
To read more please click on the link below.
Read Article
Related Articles
Blynda’s Journey with HPP
When I was born in 1968, my parents assumed everything was fine. They took me home and being her 1st child, my mom thought everything…
Living with HPP- Joann
This is me, Joann. Living with a rare disease. Hiding for years in my DNA. It snuck up on me in the prime of my…
Gene Therapy for Hypophosphatasia: Current Management and Future
To read more please click on the link below. Read Article
Patient of the Month- Emily H.
If you would like to make a donation to Soft Bones in honor of Emily, please click the button below! Donate Here Hi, this is…
Hypophosphatasia: Vitamin B6 status of affected children and adults
To read more please click on the link below. Read Article
Responses