Orphan Drug, FDA
KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
KRRO-110 is the first RNA editing development candidate from Korro's proprietary OPERA TM platform and is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD. Dosing of the first two single ascending dose cohorts in healthy adult volunteers has been completed, and an interim readout is expected in the second half of 2025.
DiagnaMed Granted FDA Orphan Drug Designation for Molecular Hydrogen to Treat Amyotrophic Lateral Sclerosis
"Receiving orphan drug designation from the FDA is a significant milestone for DiagnaMed and validates the promise of molecular hydrogen as a potential treatment for ALS. We are excited to partner with Revive Therapeutics to advance this program and meet the urgent needs of the ALS community."
In 1983, Congress passed the Orphan Drug Act (“ODA”) as part of an effort to provide market incentives for pharmaceutical manufacturers to produce “orphan drugs,” drugs for diseases that impact ...
Iconic Castro 24-hour diner Orphan Andy's (3991 17th Street) has been listed for sale after nearly 50 years of serving the ...
Versatile queen Tatiana Maslany scored a new role. Will channeling Orphan Black's iconic clone appeal to non-genre fans again ...
This week is World Orphan Week, and with it one group says the global crisis facing orphaned and vulnerable children demands ...
PEOPLE can share an exclusive clip from the March 2 episode of the series 'No Taste Like Home,' which sees host Antoni Porowski taking Justin Theroux on a journey to learn about an orphan ancestor ...
The Starfish Orphan Ministry held its semi-annual Community Free Day event, where shoppers could fill a 45-gallon bag or a ...
With orphan drugs typically being high value, the potential risk of stock losses should be a top concern for biotechs looking ...
The European orphan drug market is growing and is already comparable in size and scope to the US market. The European Union ...
The FDA granted orphan drug status to rhenium-186 obisbemeda for treating leptomeningeal metastases in lung cancer.
Fans of ultra-premium bourbon have a new toothsome tipple to chew on this week. Fanged Pursuit is a 17-year-old Kentucky ...
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