New Rare Disease Drugs and Research Advancements
XTalks
FEBRUARY 27, 2024
There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. With the approval, Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy also became the first CRISPR/Cas9-based therapy approved in the US.
59 
Let's personalize your content