Articles related to RARE DISEASES

Stiff-person syndrome, a rare yet impactful neurological disorder, presents a unique intersection with various autoimmune diseases. Understanding its symptoms and treatment strategies is crucial for patient care.

Uncover the groundbreaking advancements in PNH treatment, from oral therapies to dual inhibition strategies, and their implications for patient outcomes.

In a recent breakthrough study, thalidomide has been shown to significantly reduce recurrent bleeding in patients with small-intestinal angiodysplasia. This could pave the way for more effective treatments in the future.

A recent case study stresses the importance of considering Addison disease in patients presenting with long-standing hyperpigmentation. Notably, the patient’s potassium level was found to be 5.7 mmol/L (reference range 3.6–4.8) at admission.

This case study underscores the importance of personalized treatment in managing Mooren’s ulcer. The patient’s successful vision correction, achieved through a combination of corneal transplant, medication, and scleral lenses, highlights the potential for effective long-term management of this rare condition.

The FDA’s recent approval of Pombiliti + Opfolda just expanded treatment options for the rare inherited lysosomal disorder.