by Madaline Spencer | May 5, 2025
Dean Suhr, President and co-founder of the MLD Foundation, discusses newborn screening updates and plans for gaining recommended uniform screening panel (RUSP) approval. As noted by Mr. Suhr, the recent termination of the Advisory Committee on Heritable...
by Madaline Spencer | May 2, 2025
The U.S. Food and Drug Administration (FDA) has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis (MG). The approval is for patients, 12 years of age and older, with anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase...
by Madaline Spencer | May 1, 2025
Nicola Longo, MD, PhD, Professor and Chief of Division of Clinical Genetics at the University of California, Los Angeles, discusses data presented at the 2025 ACMG Annual Clinical Genetics Meeting highlighting the benefits of sepiapterin in patients with classical...
by Madaline Spencer | Apr 30, 2025
The U.S. Food and Drug Administration (FDA) has approved Zevaskyn (prademagene zamikeracel or pz-cel) for the treatment of wounds in patients with recessive dystrophic epidermolysis bullosa (EB). EB is a group of rare genetic skin diseases that cause the skin to...
by Madaline Spencer | Apr 29, 2025
The U.S. Food and Drug Administration (FDA) has approved nivolumab plus ipilimumab for first-line therapy in adult patients with unresectable or metastatic hepatocellular carcinoma (HCC). HCC is a rare carcinoma of the liver characterized by nodules occurring anywhere...
