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.
January 16th 2025
Axatilimab in 9 mg and 22 mg vial sizes are expected to launch in early February for the treatment of chronic graft-versus-host disease.
Is PJP Prophylaxis Indicated in Patients With Giant Cell Arteritis or Polymyalgia Rheumatica?
March 12th 2023Results of a recent study showed that the risk of pneumocystis jiroveci pneumonia (PJP) is low in patients with giant cell arteritis (GCA) and polymyalgia rheumatica (PMR), therefore discouraging the routine prescribing of PJP prophylaxis for this patient population.
AAAAI 2023 Data Identify Dysregulated Pathways in Hereditary Angioedema Prodromes and Acute Attacks
March 1st 2023Approximately 42% of prodrome-associated DEGs were also associated with hereditary angioedema attacks, indicating that multiple enriched gene networks with common hub genes and upstream regulators are shared between the prodromal and acute attack stages.
Debajyoti Ghosh, PhD: Potential Benefits of Treating Hereditary Angioedema Early Symptoms
February 27th 2023In a presentation at the AAAAI 2023 Annual Meeting, new data suggested that treating early symptoms may be a viable strategy for preventing acute attacks in patients with hereditary angioedema (HAE).
Novel Iron Supplement Effective in Treating Iron Deficiency Anemia in Children
February 21st 2023Phase 2 trial shows iron hydroxide adipate tartrate (IHAT) is non-inferior to ferrous sulfate (FeSO4) in correcting iron deficiency and hemoglobin response, with a lower incidence of moderate-severe diarrhea.
Velmanase alfa-tycv: First FDA-Approval for Non-CNS Manifestations of Alpha-Mannosidosis
February 17th 2023Velmanase alfa-tycv (Lamzede) is approved as the first and only enzyme replacement therapy for non-central nervous system manifestations of alpha-mannosidosis, an extremely rare, progressive, lysosomal storage disorder.
Weekly Efanesoctocog Alfa Improves Bleeding Prevention, Patient Health in Hemophilia A
February 15th 2023Once-weekly efanesoctocog alfa is a safe and effective treatment for patients with severe hemophilia A, providing better outcomes than current treatment options by significantly reducing bleeding rates and improving physical health, pain, and joint health.
New Data Show Severe Maternal Morbidity is a Threat to People with Sickle Cell Disease
February 2nd 2023"Despite advances in SCD management and high-risk pregnancy care, at the national level, outcomes in this population have not improved since the last NIS analysis of data from 1999 to 2008," investigators write.