Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
Articles by Patricia Inácio, PhD
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
Note: This story was updated June 16, 2021, to clarify that the HGB-207 and HGB-212 clinical trials were testing betibeglogene…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
Adding Cell Source’s special class of immune cell, Veto T-cells, to blood stem cells from an unrelated donor led…
A recent manufacturing agreement between Aruvant Sciences and Lonza will advance the development of ARU-1801, an investigational gene therapy for…
A clinical trial will assess a combination of MGTA-145 plus plerixafor as a new regimen for aiding in the collection…
Treatment with Oxbryta (voxelotor) may be a feasible alternative to blood transfusions for sickle cell disease (SCD) patients who…
FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, is entering a…
Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the…