Inborn errors of metabolism elicit unique challenges for therapy development
As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare diseases.
Therapy Area
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Why pharma is still far from a treatment for galactosemia
FDA grants fast track status to Pyxis’ PYX-201
Sanofi’s Sarclisa combo approved in Japan for newly diagnosed MM
FDA accepts BMS’ Opdivo-Yervoy combo sBLA for colorectal cancer
Semaglutide shortage ends as FDA sets deadline for compounders
WHO targets over half a million children in Gaza for polio vaccine drive
