US FDA approves IntraBio's drug for rare genetic disease
The U.S. Food and Drug Administration approved IntraBio's drug for a rare and fatal genetic disorder, the health regulator ...
Capricor: DMD Treatment Focus With Deramiocel Is Likely To Pay Off
Capricor announced it intends to file a BLA for deramiocel for treatment of patients with DMD cardiomyopathy. Click here to ...
WebMD5h
FDA OKs First Treatment for Rare, Deadly Genetic Disorder
Miplyffa treats Niemann-Pick disease type C, which progressively impacts the abilities to speak, swallow, and walk or move ...
Targeted Oncology5h
LP-184 Earns 3 FDA Rare Pediatric Disease Designations in Solid Tumors
The novel agent LP-184 has been granted rare pediatric disease designations in malignant rhabdoid tumors, rhabdomyosarcoma, ...
Researchers identify genetic mutations for rare disorder causing cognitive decline
A rare cause of hereditary cognitive decline known as CSF1R-Related Disorder (CSF1R-RD) gets its name from mutations in the ...