by Peter Ciszewski | Nov 18, 2021
Alan Mendelsohn, MD, Chief Medical Officer at Timber Pharmaceuticals, gives a detailed overview of congenital ichthyosis. As Dr. Mendelsohn explains, congenital ichthyosis is a rare genetic skin disorder characterized by dry, thickened, and scaling skin....
by Peter Ciszewski | Nov 17, 2021
The U.S. Food and Drug Administration (FDA) has approved ropeginterferon alfa-2b-njft (Besremi) for the treatment of polycythemia vera. Polycythemia vera is a rare blood disorder characterized by an increased concentration of blood cells. In most cases, polycythemia...
by Peter Ciszewski | Nov 17, 2021
Daniela Rogoff, MD, PhD, Vice President of Clinical Development at QED Therapeutics, a BridgeBio Company, discusses the phase 2 clinical trial testing infigratinib to treat achondroplasia. Achondroplasia is a skeletal dysplasia characterized by the failure of...
by Peter Ciszewski | Nov 16, 2021
Anne Pariser, MD, Director of the NCATS’ Office of Rare Diseases Research, explains why the lack of ICD codes for most rare conditions impedes diagnosis, treatment, and research. In a recent NCATS study published in the Orphanet Journal of Rare Diseases, it was...
by Peter Ciszewski | Nov 15, 2021
James Cassidy, MD, PhD, Chief Medical Officer at SpringWorks Therapeutics, discusses the ReNeu trial, which is designed to evaluate the safety, effectiveness, and tolerability of mirdametinib in neurofibromatosis type 1 (NF1) patients with inoperable plexiform...
