UConn, Connecticut Children’s trial gene-editing for rare disease
Julieta Bonvin Sallago is constantly plagued by the worry that she will not wake up in the middle…
Study Links Gout and GCKR Gene
Image Credit: Adobe Stock Results from a recent systematic review and meta-analysis of the relation between single-nucleotide polymorphism…
FDA denies Barth syndrome drug approval, suggests accelerated pathway
For the past decade, Stealth BioTherapeutics has ridden a rollercoaster trying to convince the Food and Drug Administration…
Amid FDA chaos, approval of a rare disease drug gets delayed — again
Ed Silverman, a senior writer and Pharmalot columnist at STAT, has been covering the pharmaceutical industry for nearly…
Rapamycin Improves PROMs in Patients With ME/CFS in Early Trial
Stephanie L. Grach, MD, MS Credit: Mayo Clinic Weekly rapamycin improved patient-reported outcome measures (PROMs) in patients with…