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 ...

Recurrence risk rose sharply with higher scores, with marked differences in 1- and 2-year outcomes. The preoperative circulating tumor cell-aspartate aminotransferase-to-lymphocyte ratio index ...

Test Your Knowledge About Eteplirsen for the Treatment of DMD Test Your Knowledge About the Treatment of DMD Test Your Knowledge About the Epidemiology of DMD Test Your Knowledge About the ...

Patients with atrial fibrillation or flutter were more likely to be White, male, older, and have wild-type ATTR-CM.

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

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 ...

Sharon Hesterlee, PhD, who previously served as chief research officer at the MDA, has been named interim president and CEO. Donald S. Wood, PhD, has retired from the Muscular Dystrophy Association ...

Diagnostic techniques, including dried blood spot screening in selected populations and muscle MRI, allow timely intervention and a proper evaluation of progressive muscle degeneration. Routine ...

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 ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

Sandy Siegel started the charity 30 years ago with his wife after she was diagnosed with transverse myelitis. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sandy Siegel, ...

These findings demonstrate that caregiving for a patient with HAE is a high-intensity role that severely compromises emotional well-being and productivity. The unpredictable and potentially ...