by Peter Ciszewski | Jun 27, 2023
The US Food and Drug Administration (FDA) has approved rozanolixizumab-noli (Rystiggo) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosine kinase (MuSK) antibody...
by Peter Ciszewski | Jun 22, 2023
The US Food and Drug Administration (FDA) has approved the gene therapy, delandistrogene moxeparvovec (Elevidys), to treat patients ages 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. DMD is a severe progressive...
by Madaline Spencer and Peter Ciszewski | Jun 15, 2023
Pierantonio Russo, MD, Chief Medical Officer at Eversana, discusses the expanding role artificial intelligence (AI) plays in the diagnostics of rare conditions. As noted by Dr. Russo, diagnostic delays are expected in rare diseases due to many factors....
by Peter Ciszewski | Jun 13, 2023
The U.S. Food and Drug Administration (FDA) has approved odevixibat (Bylvay) to treat cholestatic pruritus in patients from 12 months of age with Alagille syndrome. In 2021, the FDA approved maralixibat (Livmarli) to treat cholestatic pruritus in patients from 12...
by Peter Ciszewski | May 22, 2023
The US Food and Drug Administration (FDA) has approved avapritinib (Ayvakit) for the treatment of adults with indolent systemic mastocytosis. Systemic mastocytosis is a rare disease, usually caused by mutations the KIT D816V gene. The disorder is characterized by...
