Why Rare Disease Patients Should Worry About the Shifting Discussion from Value to Scarcity

The high cost of rare disease therapies is often…

Albireo Enrolls First Patient in Late-Stage Study of Treatment for Life-Threatening Pediatric Liver Disorder
Rare Daily Staff Albireo Pharma said it enrolled the first patient in a late-stage clinical trial of its lead product candidate A4250, an… Continue Reading
FDA Grants Castle Creek Rare Pediatric Disease Designation for EB Therapy
  Rare Daily Staff The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Castle Creek Pharmaceuticals’… Continue Reading
FDA Grants Myonexus Therapeutics Rare Pediatric Disease Designation
Rare Daily Staff The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Myonexus Therapeutics’ MYO-101, an… Continue Reading
Eiger Expands License Agreement with Merck, Enters Agreement with Progeria Research Foundation
Rare Daily Staff Eiger BioPharmaceuticals said it has expanded its licensing agreement with Merck to include rights to develop the… Continue Reading
FDA Approves Subcutaneous Actemra for Children with PJIA
Rare Daily Staff The U.S. Food and Drug Administration approved the subcutaneous formulation of Genentech’s Actemra for the treatment in… Continue Reading
Gene Editing Company Beam Therapeutics Launches with $87 Million Round
Rare Daily Staff Beam Therapeutics, a gene editing company founded by leading scientists in CRISPR gene editing, today announced its launch… Continue Reading
Abeona Expands Board with Two Appointments
Rare Daily staff Cell and gene therapy developer Abeona Therapeutics that it is expanding its board with the addition of two independent… Continue Reading
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