Rare Leader: Vanessa Vogel-Farley Executive Director Dup15q Alliance

The Basics Name:  Vanessa Vogel-Farley Title:…

BridgeBio Enters Collaboration with Cincinnati Children’s
Rare Daily Staff BridgeBio Pharma and Cincinnati Children’s Hospital Medical Center said they are entering into a research collaboration… Continue Reading
Myonexus Therapeutics Initiates First-in-Human Trial of Gene Therapy for Rare Type of Muscular Dystrophy
Rare Daily Staff Myonexus Therapeutics, a gene therapy company developing first-ever corrective gene therapies for limb girdle muscular… Continue Reading
Phoenix Nest Licenses Sanfilippo Syndrome Gene Therapy Prepares Clinical Trial
Rare Daily Staff Phoenix Nest exclusively licensed a gene therapy for Sanfilippo syndrome (also known as Mucopolysaccharidosis IIIC, (MPS… Continue Reading
The Potentially Deadly Consequences of an Oversight in Newborn Screening Tests
When Jon Miller’s son was born doctors failed to diagnose a rare and deadly medical condition despite using newborn screening that was… Continue Reading
Cure SMA Launches New Support Program for Teens and Adults with SMA
Rare Daily Staff  Besides funding SMA research and care, Cure SMA provides thousands of individuals and families with vital family support… Continue Reading
Rare Leader: Rachelle Dixon, President and Co-Founder, HSAN1E Society
The Basics Name:  Rachelle Dixon Title:  President and co-founder Organization: HSAN1E Society Disease focus: Hereditary sensory and… Continue Reading
Mirum Pharmaceuticals Launches with $120 Million in Series A Financing to Develop Maralixibat for Rare Liver Diseases
Rare Daily Staff Mirum Pharmaceuticals has launched with $120 million in series A financing to support development of its lead drug… Continue Reading
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