Articles related to RARE DISEASES

Explore the intricacies of diagnosing and treating acral melanoma, a subtype that presents unique challenges but also offers new avenues for targeted therapies and immune modulation.

This phase 1–2 study of gene therapy in Crigler–Najjar syndrome patients offers promising insights into a novel treatment approach. Explore the detailed findings to understand how this therapy could revolutionize the management of this rare and severe condition.

Home-based therapies are emerging as a viable solution to the challenges faced by global healthcare systems, particularly for conditions like sickle cell disease. Dive deeper into the research and implications for patient care.

Explore the unmasking of postural orthostatic tachycardia syndrome (POTS) amid the COVID-19 pandemic and its vaccine era, with the integration of traditional and alternative treatments offering renewed hope for affected patients.

Gain insight into how a shift in cellular environment and UV radiation exposure can instigate additional mutations, deepening our understanding of the evolution and development of rare and complex malignancies such as BPDCN.

The Low-PV phase 2 trial provides compelling evidence for the use of ropeginterferon alfa-2b in the management of low-risk Polycythemia vera, demonstrating its superiority over the standard treatment of therapeutic phlebotomy and aspirin in maintaining hematocrit levels and reducing disease-related symptoms.