The FDA grants orphan drug designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 ... Dec 30
AP103 is based on Amryt's gene-therapy platform technology and offers a potential treatment for patients with DEB... Dec 24
UX701 is currently in late preclinical development, and an investigational new drug (IND) application is expected to be filed with the US FDA by the ... Dec 11
HBI-8000 for Monotherapy Adult T-cell Leukemia-Lymphoma ATL ... Oct 01
Zynerba Pharmaceuticals receives orphan drug designation for cannabidiol for the treatment of 22q11.2 deletion syndrome ... Sep 18
... Sep 08
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