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
To read more please click on the link below.
Read Article
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…
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…
Not long ago, Becky R. and her husband adopted a little baby girl, Mei Mei, from China. When she was first adopted, doctors were worried…
My name is Cami Rush and I am the mother to three children. I have an 18 year old son, Connor (diagnosed with HPP around 18…
Hi, we are twin sisters Amy and Suzanne, we have been dealing with symptoms of HPP for a lifetime. Our diagnosis seemed as though it…
The US Hypophosphatasia Foundation
141 Hawkins Place, #267
Boonton, NJ 07005, USA.
Organization
Programs
Community
Responses