AI model identifies existing drugs that can be repurposed for treatment of rare diseases
There are more than 7,000 rare and undiagnosed diseases globally. Although each condition occurs in a small number of ...
How do rare genetic variants affect health? AI provides more accurate predictions
Whether we are predisposed to particular diseases depends to a large extent on the countless variants in our genome. However, ...
Neurolixis Secures Orphan Medicinal Product Designation For NLX-112 As A Treatment For Spinocerebellar Ataxia
European Commission approves OMP Designation for NLX-112 (befiradol) following compelling results in studies funded by the US Dept of De ...
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 ...
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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 ...
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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, ...