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 ...
WebMD14h
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 Oncology14h
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, ...
Sutro Biopharma's Competitive ADCs Position It For Future Growth In Oncology
Sutro Biopharma specializes in ADCs for oncology and shows promise with STRO-002 for ovarian cancer and collaborations with ...
Lantern Pharma Gets Three FDA Rare-Pediatric Designations for LP-184
Lantern Pharma has won a trio of rare-pediatric-disease designations from the Food and Drug Administration for its investigational drug candidate LP-184 in multiple ultra-rare children's cancers.
Lantern Pharma Announces Three U.S. FDA Rare Pediatric Disease Designations Granted to LP-184 in Multiple Ultra Rare Children’s Cancers
Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence ("AI") company developing targeted and transformative cancer ...
GEN1d
StockWatch: FDA Surprise Lifts Expectations for Applied’s Govorestat
Investors and analysts predict the FDA will approve Applied Therapeutics' lead candidate govorestat for classic galactosemia ...
Kazia Therapeutics Announces Presentation of EVT801 Clinical Data at 15th Biennial Ovarian Cancer Research Symposium
Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company, is pleased to announce the ...