Articles
A Find-and-Replace Approach to Fixing the Mutation Underlying Sickle Cell Disease
Read More
Bluebird Withdraws Gene Therapies in Europe as it Winds Down Operations There
Read More
Rare Leader: Erica Webster, Co-Founder and President, Hope for Hypothalamic Hamartomas
Read More
Crinetics Raises $150 Million in Public Offering to Advance Rare Endocrine Disease Pipeline
Read More
ReCode Raises $80 Million to Advance Pipeline of Rare Lung Diseases Therapies
Read More
FDA Awards Castle Creek $1.8 Million Grant to Support Pivotal Study in RDEB
Read More
Selecta and Genovis Enter License Agreement to Advance Next-Generation IgG Protease in Gene Therapy and Autoimmune...
Read More
Study Finds Potential to Repurpose Cancer Therapies to Treat Rare Diseases
Read MoreNews
Social
RT @CFUCDF: Help us reach our goal🦓❤️@HorizonNews @ultragenyx @GlobalGenes @EveryLifeOrg @remember_girls @VitafloUSA @nord @Tr…
RT @GlobalGenes: “The organization is driven by the frustration that a lot of rare disease families...
RT @GlobalGenes: Podcaster, author and an inspiration to many. Hear how @KyleABryant’s cross-country bike ride changed...
