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.
October 25th 2024
On October 25, 2024, Kind Pharmaceutical announced receipt of FDA Orphan Drug Designation for AND017 in the treatment of sickle cell disease.
A Hidden Crisis: The Need for Treatments in Notalgia Paresthetica
March 28th 2023A silent disease characterized as a chronic cutaneous neuropathy, notalgia paresthetica has an unmet need yet to be fully understood and, dermatologists argue, an FDA-approved treatment could help improve understanding of the etiology and prevalence of this disease.
Revolo Readying Data on Allergen Sensitivity Drug
The treatment is a first-in-class peptide derived from mTB Chaperonin 60.1 that is often involved in resetting the immune system. The treatment is being evaluated in patients with eosinophilic esophagitis and allergen sensitivity.
Leniolisib Becomes First FDA-Approved Treatment for Rare Immunodeficiency Disease
March 24th 2023Phase 2/3 data show significant improvement in lymphoproliferation as measured by the reduction in lymph node size and increase in naïve B cells in patients with activated phosphoinositide 3-kinase delta syndrome.
New Biomarkers Predict Adverse Events in Cardiac Sarcoidosis
March 23rd 2023Data from a meta-analysis show late gadolinium enhancement (LGE) on cardiac MRI scans and elevated fluorodeoxyglucose (FDG) uptake on PET scans are both strong predictors of major adverse cardiac events for cardiac sarcoidosis.
Margaret Ragni, MD, MPH: Impact of new Antihemophilic Factor VIII Therapy on Hemophilia A
March 15th 2023After a year of treatment with once-weekly antihemophilic factor (recombinant), Fc-VWF-XTEN Fusion Protein-ehtl, results show patient pain was significantly reduced and quality of life significantly increased.
Knowledge of Severity, Susceptibility Shape Perceptions of Sickle Cell Trait Testing
March 13th 2023Findings from the study emphasize the importance of disseminating knowledge and education to reduce disease burden after examining healthcare students' knowledge and attitudes towards sickle cell trait (SCT) screening in Ghana.
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.