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...
by Peter Ciszewski | Sep 28, 2023
Pulmonary arterial hypertension (PAH) is a rare and progressive disease characterized by the narrowing of blood vessels in the lungs, leading to increased strain on the heart. Merck has developed a potential breakthrough treatment for PAH called sotatercept. PAH is a...
