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 ...

Patients with hereditary ATTR-CM were younger, had better myocardial function, and had higher arterial stiffness compared with patients with wild-type ATTR-CM. The type of mutation in the ...

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

Ambulatory boys with DMD treated with delandistrogene moxeparvovec maintained North Star Ambulatory Assessment scores above baseline at year 3. Sarepta Therapeutics announced positive 3-year ...

An IDEAL-IQ FF threshold of 52.3% in the vastus lateralis accurately identified loss of ambulation with 92.5% sensitivity and 100% specificity. Findings from a recent study published in the Journal of ...

Tafamidis clinically stabilized neuropathy in most patients who developed acquired transthyretin amyloidosis following a domino liver transplantation. Patients who receive livers from individuals with ...

Patients with MG who took C5 inhibitors saw greater reductions in concomitant OCS and NSIST use than did those who took efgartigimod. Rare Disease Advisor, a trusted source of medical news and feature ...

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 ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

The US Food and Drug Administration (FDA) has cleared AnemoCheck Home for patients with sickle cell disease (SCD), iron deficiency anemia, vitamin B12 deficiency anemia, folate deficiency anemia, and ...

The recommendations were based on the available evidence, its certainty, and key judgments using the Grading of Recommendations Assessment, Development and Evaluation framework. New guidelines ...

Speakers at the panel “Unlocking the future: the convergence of AI, personalized medicine, and genomics” at the World Orphan Drug Congress USA 2025 (Photo by Riya Ajmera) Artificial intelligence could ...