by Peter Ciszewski | Jan 4, 2023
Ankit Mehta, MD, Nephrologist and Program Director for the Department of Internal Medicine at Baylor University Medical Center, discusses the current treatment landscape for Fabry disease, including approved and investigational therapies. Fabry disease is a...
by Peter Ciszewski | Jan 3, 2023
2025 Orphan Drugs: PDUFA Dates and FDA Approvals 2024 Orphan Drugs: PDUFA Dates and FDA Approvals 2023 Orphan Drugs: PDUFA Dates and FDA Approvals 2022 Orphan Drugs: PDUFA Dates and FDA Approvals 2021 Orphan Drugs: PDUFA Dates and FDA Approvals Rare diseases and...
by Peter Ciszewski | Jan 3, 2023
Morie Gertz, MD, Hematologist, Chair Emeritus Internal Medicine, Mayo Clinic, discusses post hoc analysis data on the survival benefit of birtamimab in high-risk amyloid light chain (AL) amyloidosis in the phase 3 VITAL study. These data were recently presented...
by Peter Ciszewski | Dec 29, 2022
Jeffrey L. Neul, MD, PhD, Director of the Vanderbilt Kennedy Center, and Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, at the Vanderbilt University Medical Center, discusses disorders related to Rett syndrome that are also...
by Peter Ciszewski | Dec 23, 2022
Ying Huang, PhD, CEO of Legend Biotech, discusses updated results from the CARTIFAN-1 study, evaluating cilta-cel in patients in China with relapsed/refractory multiple myeloma who have received ≥3 prior lines of therapy. These data were recently...
