The U.S. Food and Drug Administration (FDA) has accepted Ultragenyx Pharmaceutical‘s application seeking approval of UX111, a gene therapy…
Articles by Patricia Inácio, PhD
Note: This story was updated Dec. 11, 2023, to correct that Kyowa Kirin has entered into an agreement to acquire…
The 2021 Race Against Dementia post-doctoral fellowship has been awarded to scientist Karissa Barthelson, PhD, to investigate the common pathways underlying…
A 30-minute, inexpensive, and easy-to-use test may help measure the activity of NDST1, one of the main enzymes involved in…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
The U.S.’ largest pilot newborn screening program for multiple disorders, including Sanfilippo syndrome type A and type B, has…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
Following the promising results of a second-generation gene therapy in a mouse model of Sanfilippo syndrome type A, a…
Preliminary results from the first patient treated with Orchard Therapeutic’s OTL-201 show that the…