Danicopan Approved as Add-On Therapy for PNH Treatment

Ultomiris (ravulizumab) and Soliris (eculizumab), both developed by global biopharmaceutical leader AstraZeneca, are both effective treatments for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Yet this efficacy…

Continue Reading Danicopan Approved as Add-On Therapy for PNH Treatment

Rare Community Profiles: Kristi’s Fight to Combat Misconceptions about Prader-Willi Syndrome (PWS): “Our Children Have PWS; They Are Not PWS”

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Continue Reading Rare Community Profiles: Kristi’s Fight to Combat Misconceptions about Prader-Willi Syndrome (PWS): “Our Children Have PWS; They Are Not PWS”