Measuring the Value of Therapies for Ultra-Rare Diseases

  The pricing of drugs is characterized by a…

Sangamo Treats First First Patient in Genome Editing Trial for MPS II
Rare Daily Staff Sangamo Therapeutics said it treated the first patient in the phase 1/2 clinical trial of its experimental in vivo genome… Continue Reading
Ultragenyx Completes Dosing of First Cohort in Study of Gene Therapy in OTC Deficiency
Rare Daily Staff Ultragenyx Pharmaceutical said it has completed patient dosing in the first cohort of three patients enrolled in a phase… Continue Reading
Sobi Initiates Late-Stage Study of Kineret to Treat Still’s Disease
Rare Daily Staff Swedish Orphan Biovitrum said that it has randomized the first patient in its late-stage study of Kineret in the treatment… Continue Reading
Rare Leader: Debra Miller, CEO and Founder of Cure Duchenne
Name: Debra Miller Title: CEO and founder Organization: Cure Duchenne Disease focus: Duchenne Muscular Dystrophy Headquarters: Newport… Continue Reading
Ultragenyx Wins FDA Approval of First Therapy for MPS VII
Rare Daily Staff The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical’s MEPSEVII, the first treatment for children and… Continue Reading
Naia Rare Diseases Gets Green Light for Clinical Trials of Treatment for Short Bowel Syndrome
Rare Daily Staff Naia Rare Diseases, a biopharmaceutical company developing drugs for rare gastrointestinal diseases, said it has received… Continue Reading
Inozyme Pharma Raises $49 Million to Develop Therapies for Rare Diseases Affecting Soft Tissue and Bone
Rare Daily Staff Inozyme Pharma, a drug developer focusing on rare diseases involving calcification of soft tissues and bone, completed a… Continue Reading