How the Orphan Drug Act Has Changed the Rare Disease Landscape | CheckRare.com
Badhiu Basu, PhD, owner of the Converge Advisory Group talked about the Orphan Drug Act and what it has (and has not) accomplished.
Badhiu Basu, PhD, owner of the Converge Advisory Group talked about the Orphan Drug Act and what it has (and has not) accomplished.
Marie Abrego, patient with NMOSD, and Wendy Erler, VP of Patient Experience at Alexion, discuss NMOSD diagnosis and finding support.
Diego Ferone, MD, PhD, Endocrine Unit Chief at San Martino Hospital, discusses a novel octreotide formulation for the treatment of acromegaly.
For older patients with acute myeloid leukemia undergoing allo-HCT, posttransplant outcomes have improved over time.
Nicholas Silvestri, MD, of the Univ of Buffalo, provides an overview of the latest myasthenia gravis clinical research presented at AAN 2024.
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the nervous and musculoskeletal systems of young patients.
Diane Schriner, Vice President of Erdheim-Chester Disease Global Alliance, provides an overview of Erdheim-Chester disease (ECD).
Michelle Reyes, patient and AD of the HypoPARAthyroidism Association, discusses living with the disease and how the organization can help.
Leslie Edwin, CSRF President, and Gretchen Jordan, CSRF Associate Director, discuss how their organization supports patients with Cushing’s disease/syndrome.
The first-in-class oral factor B inhibitor iptacopan improves hematologic and clinical outcomes in anti-C5-treated patients with persistent anemia and in those who have not received…
Male and female p.C282Y homozygotes, including those undiagnosed with hemochromatosis, have increased morbidity risks.