12.11.2018 When Jessica Klein-Foglio’s and Michael Foglio’s son Ben was diagnosed in 2017 with Salla disease, an ultra-rare lysosomal disorder, the… Continue Reading
12.10.2018 Rare Daily Staff The Castleman Disease Collaborative Network and Medidata said they discovered new Castleman patient subgroups, based on… Continue Reading
12.7.2018 Rare Daily Staff Orchard Therapeutics said it presented the first demonstration that its experimental gene therapy OTL-102 for the treatment… Continue Reading
12.7.2018 The U.S. Food and Drug Administration recently granted gene therapy developer RegenxBio Orphan Drug designation for its experimental gene… Continue Reading
12.6.2018 Rare Daily Staff The 100,000 Genomes Project, led by Genomics England in partnership with NHS England, has reached its goal of sequencing… Continue Reading
11.27.2018 Rare Daily Staff The Ireland Strategic Investment Fund, WuXi NextCODE, and Irish life sciences company Genomics Medicine Ireland outlined a… Continue Reading
11.27.2018 Earlier this month during Novartis’ R&D Day, Dave Lennon caught the attention of the payer community when he said that the company’s… Continue Reading
