by Madaline Spencer | Aug 22, 2026
The US Food and Drug Administration (FDA) has approved Pasatru (garetosmab-grts) to reduce formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP). FOP is a disorder in which...
by Madaline Spencer | Aug 21, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, in patients eight years and older with glycogen storage disease type 1a (GSD1a). GSD1a is a rare metabolic disorder that...
by Madaline Spencer | Aug 21, 2026
Byron Wade, a patient with IgA nephropathy (IgAN), discusses his personal diagnostic journey and a recent study looking at other patients’ experiences. IgAN is characterized by the accumulation of IgA antibodies in the kidneys. In the early stages,...
by Madaline Spencer | Aug 19, 2026
Matt Trudeau, President of ITF Therapeutics, discusses the importance of patient listening in the development of Duvyzat (givinostat) for Duchenne muscular dystrophy (DMD). DMD is a rare genetic, neuromuscular condition characterized by progressive...
by Madaline Spencer | Aug 18, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval to iberdomide (Zenbexus) in combination with daratumumab and hyaluronidase-fihj and dexamethasone for adults with multiple myeloma (MM) who have received at least one prior line of therapy...
