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 ...

Nearly two-thirds of adult patients on crinecerfont achieved glucocorticoid doses within the physiologic range, compared to just 18% on placebo. The US Food and Drug Administration (FDA) has approved ...

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 ...

Dana Rizk, MD, professor of medicine in nephrology at the University of Alabama at Birmingham Heersink School of Medicine, speaks at the Renal Physicians Association 2026 Annual Meeting in Atlanta, ...

A new capsid improves skeletal and cardiac muscle tropism, reducing off-target effects and enabling rapid transduction and transgene expression. New strategies to overcome immune responses against ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Miller, MD, a professor of pediatric endocrinology at the University of Florida in Gainesville who specializes in ...

The CAPTIVATE trial met its target of at least 20 responders among the first 40 participants before all 40 completed part A. Dianthus Therapeutics announced an early “go” decision for claseprubart for ...

CAD accounts for approximately 15% of AIHA cases 2; various sources indicate that anywhere from 7% to 25% of cases of AIHA are caused by CAD. 3 Secondary CAD occurs as a result of underlying ...

The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with Duchenne muscular dystrophy (DMD), according to a new study ...

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...

Routine radiological imaging, along with clinical evaluation, may reveal pathological TLK early enough to provide corrective measures. Thoracolumbar kyphosis (TLK) is common in children with ...

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 ...