Why a Sports Physical May Trigger a Doctor to Think About a Rare Diagnosis

Marfan syndrome is a genetic disorder that affects…

FDA Grants Zogenix Breakthrough Therapy Designation for Experimental Dravet Syndrome Therapy
Rare Daily Staff The U.S. Food and Drug Administration awarded Zogenix Breakthrough Therapy Designation for its investigational product,… Continue Reading
Patient Groups Call on House to Reject Right to Try Act
Rare Daily Staff The National Organization for Rare Disorders along with 37 patient and provider organizations in a letter to leaders of the… Continue Reading
A Rare Cancer in Need of Congressional Recognition
Sarcoma has long been under-represented in the research landscape, in part because it represents just 1 percent of all cancers. There are… Continue Reading
How Patients Can Accelerate Translational Research in Rare Disease
World Rare Disease Day is marked on the last day of February by rare disease patient advocates across the globe as a way to raise awareness.… Continue Reading
Global Genes Rare Disease Stock Index Falls 3.84% for the Week as Stock Have Worst Week in Two Years
Stocks tumbled on Friday in the single biggest percentage decline in the Dow Jones Industrial Average since June 2016 and its worst weekly… Continue Reading
Rare Leader: Anne Bruns, Sponsor Chair of Utah Rare
The Basics Name: Anne Bruns Title: Sponsor Chair Organization: Utah Rare Disease focus: Coalition for rare disease organizations and… Continue Reading
Gene Therapy Developer Avrobio Closes $60 Million Financing
Rare Daily Staff Gene therapy developer Avrobio said that it has completed a $60 million series B financing, which will be used to advance… Continue Reading