FDA Commissioner Scott Gottlieb, MD, announced the agency’s progress in streamlining the orphan drug review process and outlined plans to close a loophole currently hindering pediatric drug research.

The SIS-101-ADO combines an siRNA that suppress the expression of CLCN7, with SiSaf’s Bio-Courier® next generation silicon stabilized hybrid lipid nanoparticles (sshLNP) technology that addresses the ...

But lucrative financial incentives created by the Orphan Drug Act signed into law by President Ronald Reagan in 1983 succeeded far beyond anyone’s expectations. More than 200 companies have brought ...

TORONTO and HAIFA, Israel, Feb. 02, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (NRX.V), known as "NurExone," is pleased to announce the initiation of the Orphan Drug ...

More than 30 years ago, Congress overwhelmingly passed a landmark health bill aimed at motivating pharmaceutical companies to develop new drugs for people whose rare diseases had been ignored. By the ...

CDMOs offer expertise and customization options for sponsors of orphan drugs. To foster the development of treatments for undertreated and rare conditions, FDA grants orphan drug designation to drugs ...

SiSaf’s Innovative RNA Therapeutic for Rare Genetic Skeletal Disorders Begins the U.S. Regulatory Process for Orphan Drug Designation GUILDFORD, England – 12 October 2022 – SiSaf Ltd, an RNA delivery ...