Current Status of Newborn Screening Worldwide was updated on August 31, 2016.
Global Genes has announced the 2016 RARE Champion of Hope Honourees!
The April 2106 edition of Nature Biotechnology had an article by Emil D Kakkis, Sara Kowalcyk & Max G Bronstein titled “Accessing the accelerated approval pathway for rare disease therapeutics“. It is relevant to the struggles we have seen in Canada with getting treatments for PKU reviewed and covered in a timely fashion.
PKU & ALLIED DISORDERS
European Guidelines on treatment of PKU