The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
May 10th 2024
A Veterans Affairs study finds low alpha-1 antitrypsin phenotyping rates, indicating the need for improved healthcare provider education on AATD.
Neutrophil, Platelet Engraftment is Successful with Briquilimab in Patients with Sickle Cell Disease
January 3rd 2023The targeted patient population included those with sickle cell disease or beta thalassemia who were considered at high risk for complications, or otherwise ineligible for standard myeloablative hematopoietic stem cell transplant.
From Early Hypothesis to Clinical Data About Reducing Vaso-Occlusive Episodes with Arginine Therapy
December 22nd 2022Almost 25 years later, Dr. Claudia Morris concluded 2022 by presenting phase 2 data at the 64th ASH Annual Meeting that demonstrated the therapy's benefit, and her original premise. Her team's phase 3 trial evaluating Arg is currently underway.
Corinna Schultz, MD: Does Sickle Cell Trait get Addressed?
December 12th 2022"There's no national guidance on how this information should be delivered back to families, which leaves it up to each state's newborn screening program to decide the best route that they see fit," Dr. Corinna Schultz explains.