This is just the beginning! Our collection holds over 300 years of history, including rare newspapers, obituaries, marriage records, birth records, and so much more—most of which you won’t find anywhere else!
Gene therapy offers hope yet access remains scarce for rare diseases
Robin Alderman fights for a cure for her son Camden, diagnosed with Wiskott Aldrich syndrome. Orchard Therapeutics pulled back on related therapies, leaving families to fundraise as researchers seek nonprofit routes to broaden access and Cut red tape. The rare disease burden, high costs, and slim profits deter investment, risking progress in a field with potential to help thousands worldwide.
MENTIONED IN THIS ARTICLE
Robin Alderman
Camden
Orchard Therapeutics
Alderman
Giulio Cossa
University of Manchester
England
Fighting Blindness
Opus Genetics
University of Pennsylvania
Jean Bennett
Bennett
Aldermans
Camden Alderman
Wiskott-Aldrich
U.S
North Carolina Agricultural and Technical State University
Priya Stephen
Italy
Stephen
Genethon
Frédéric Revah
GlaxoSmith-Kline
Orchard
U.S. Food and Drug Administration
Bobby Gaspar
Revah
Claire Booth
University College London
Access
Europe
Foundation For the National Institutes of Health
FDA
NIH
Anna Greka
Broad Institute
MIT
Harvard
Greka
Greensboro
N.C
Associated Press