by Peter Ciszewski | Jan 7, 2024
Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all...
by Peter Ciszewski | Dec 27, 2023
Cat Lutz, Ph.D., M.B.A. Vice President of the Rare Disease Translational Center at The Jackson Laboratory, talks about how you can help family and loved ones who suffer from rare diseases. Transcription We refer to this phase and the patients as their...
by Peter Ciszewski | Dec 20, 2023
The U.S. Food and Drug Administration (FDA) approved Filsuvez (birch triterpenes) topical gel for the treatment of partial thickness wounds in patients 6 months and older with Junctional Epidermolysis Bullosa (JEB) and Dystrophic Epidermolysis Bullosa (DEB). Filsuvez...
by Peter Ciszewski | Oct 6, 2023
The U.S. Food and Drug Administration (FDA) has recently granted Nexcella’s NXC-201 Orphan Drug Designation (ODD) for the treatment of AL amyloidosis. This designation provides certain benefits to support the clinical development of the drug, including financial...
by Peter Ciszewski | Sep 29, 2023
The U.S. Food and Drug Administration (FDA) has approved the combination of cipaglucosidase alfa-atga plus miglustat (Pombiliti+ Opfolda) to treat adults with late-onset Pompe disease. More specifically, the treatment is approved to treat those adults weighing more...
