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 ...

Between 2001 to 2011, annual age-adjusted incidence increased from 0.55/100,000 to 0.78/100,000, demonstrating an increasing trend with age. Incidence peaks in individuals over 70 years of age ...

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 ...

The updated AABB/ICTMG guideline hopes to decrease excessive platelet transfusions. The adoption of an updated, more restrictive 2025 Association for the Advancement ...

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 ...

Notably, the CRL did not identify safety or efficacy concerns. Aquestive Therapeutics, Inc. announced that the US Food and Drug Administration (FDA) has issued a complete response letter (CRL) for ...

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 ...

Obstructive sleep apnea prevalence increased in children with achondroplasia beginning at school age (6-12 years) and remained elevated into adolescence. Recurrent otitis media may accompany ...

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 ...

At present, no validated tools or scoring systems have been developed to predict prognosis or clinical course of warm autoimmune hemolytic anemia (AIHA). Several potential prognostic risk factors that ...

Nintedanib significantly reduced the decline in the forced vital capacity of patients with progressive pulmonary fibrosis (PPF). Treatment with nintedanib significantly slows the progression of ...