Racial and ethnic inequities persist in MG severity and outcomes, with a particularly high disease burden among Indigenous patients. Rare Disease Advisor, a trusted source of medical news and feature ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

HDFN can cause adverse outcomes both prenatally and postnatally. Hemolytic disease of the fetus and newborn (HDFN) can be associated with both severe pre- and postnatal outcomes, according to a study ...

Life expectancy in DMD has improved over time. Previous estimates pointed to a life expectancy of 25 years, with more recent data reporting a life expectancy of 31.7 years (95% CI, 27.4-36.0). 3,4 A ...

Nearly half of those with preexisting atrial fibrillation experienced stroke despite appropriate anticoagulation. The prevalence of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in elderly ...

New research suggests that caffeine could help treat fatigue, among the most troubling symptoms of multiple sclerosis (MS). According to the results of a small study published in the February 2026 ...

Multivariate analysis revealed several factors that affected overall survival, including a diagnosis of NF1, evidence of metastasis at diagnosis, the maximum diameter of the primary tumor at diagnosis ...

Venous thrombosis occurred in 53 patients, with a 1-year cumulative incidence of 2.6% and a 5-year cumulative incidence of 8.6%. Researchers found that promptly controlling immune thrombocytopenia ...

Unexplained anemia and hyperbilirubinemia in a patient with syncope prompted further evaluation, leading to the diagnosis of warm AIHA associated with RSV infection. A young woman presenting to the ...

The project addresses a long-standing gap in the field: the lack of a clear human reference standard for muscle regeneration. A new research collaboration between muscular dystrophy organizations aims ...

Chimeric antigen receptor (CAR) T cells based on the proximal region of mesothelin completely eradicated CCA tumors in 3 different mouse models. Mesothelin, a tumor differentiation antigen that is ...

The authors argue that the long-held label of "uniformly lethal" may no longer be appropriate and can inadvertently bias clinical decision-making. Homozygous achondroplasia is traditionally ...