by Madaline Spencer | Jun 9, 2026
A study published in the Journal of Clinical Lipidology evaluated maternal and neonatal outcomes of chenodeoxycholic acid (CDCA) treatment during gestation in women with cerebrotendinous xanthomatosis (CTX). CTX is a rare lipid storage disease characterized by the...
by Madaline Spencer | Jun 8, 2026
A study published in The Journal of Pediatrics describes a post hoc analysis of clinical trials evaluating maralixibat in children with Alagille syndrome and its effect on xanthoma severity. Alagille syndrome is a rare genetic disease that affects the liver and other...
by Madaline Spencer | Jun 5, 2026
The US Food and Drug Administration (FDA) has approved Cavhanza (nilotinib) orally disintegrating tablets for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML). CML is a myeloproliferative neoplasm characterized by the expression of...
by Madaline Spencer | Jun 5, 2026
James Howard Jr., MD, Professor of Neurology at the University of North Carolina at Chapel Hill, discusses results from the ADAPT SERON clinical trial of efgartigimod IV in patients with seronegative generalized myasthenia gravis (gMG). Data from this trial was...
by Madaline Spencer | Jun 4, 2026
Michael Levy, MD, PhD, Neuroimmunologist and Associate Professor at Harvard Medical School, discusses results from the METEOROID clinical trial of Enspryng (satralizumab) in patients with myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). ...
