Tag: RARECast

A total of 91 posts are filed under RARECast
RARECast: Accelerating Rare Disease Drug Discovery
For many rare disease patients, the drug discovery and development process moves slower than the diseases they are battling. Earlier this… Continue Reading
RARECast: How Rare Disease Patients Can Get their Voices Heard in Drug Discovery and Development
  There is increasing recognition of the important role patients can play by providing their insights into the drug discovery and… Continue Reading
RARECast: One Patient’s Difficult Road to a Rare Diagnosis
Porphyria is a rare and intensely painful blood disease. Because it can manifest itself with symptoms similar to those caused by far more… Continue Reading
RARECast: How Researchers Diagnosed Four Patients with a Never-Before-Identified Rare Disease in a Day
  Diagnosing a rare disease can take years. When it’s a disease that’s never been identified before, the search for a diagnosis can be… Continue Reading
RARECast: Stem Cell Gene Therapy Restores Immune System in Kids with SCID
Researchers at UCLA have developed a stem cell gene therapy treatment for children born with the rare, life-threatening condition… Continue Reading
RARECast: Non-Profit Drug Company Seeks to Advance Rare Disease Therapies
  The high cost of drug development and the small populations for individual rare diseases can make it difficult to attract drug companies… Continue Reading
RARECast: A Quest for Tribe: Searching for Others with the Same Ultra-Rare Mutation
When Milo Lorentzen was born, concerns over his condition sent him to a neonatal intensive care unit where he spent the first ten days of… Continue Reading
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