by Madaline Spencer | Oct 24, 2025
Lawrence Lustig, MD, Professor at Columbia University Medical Center, discusses early results from the CHORD clinical trial testing gene therapy to treat otoferlin-related hearing loss. Otoferlin (OTOF)-related hearing loss is a rare genetic auditory...
by Madaline Spencer | Oct 23, 2025
The U.S. Food and Drug Administration (FDA) has approved Epioxa (riboflavin 5’-phosphate ophthalmic solution) for the treatment of patients with keratoconus. Keratoconus is a rare eye condition characterized by the progressive thinning and weakening of the cornea....
by Madaline Spencer | Oct 23, 2025
Tiffany Graham Charkosky, author and patient with Lynch syndrome, discusses her experience with genetic testing and her book LIVING PROOF: How Love Defied Genetic Legacy. Lynch syndrome is a rare inherited condition characterized by increased risk for...
by Madaline Spencer | Oct 22, 2025
The U.S. Food and Drug Administration (FDA) has approved Gazyva/Gazyvaro (obinutuzumab) for the treatment of adults with active lupus nephritis who are receiving standard of care. In addition, the approval includes a shorter 90-minute infusion time after the first...
by Madaline Spencer | Oct 20, 2025
Christelle Huguet, PhD, Head of Research and Development at Ipsen, discusses the company’s current approved orphan drugs and those in development for rare diseases. Ipsen’s work in the rare disease space aims to address areas of high unmet medical needs;...
