Cardiac scintigraphy had higher sensitivity and specificity across different radiotracers in diagnosing cardiac amyloidosis compared to cardiac magnetic resonance imaging. Cardiac scintigraphy, ...

Clinical practice guidelines for the diagnosis and treatment of progressive pulmonary fibrosis (PPF) in adults were published in 2022 through collaboration among the American Thoracic Society (ATS), ...

The vast capabilities of artificial intelligence mean that it can applied to various domains of medicine, including in the rare disease space. To much of the public, artificial intelligence (AI) may ...

An ongoing open-label extension study suggests that the therapy's benefits may extend through at least 52 weeks. Deupirfenidone (LYT-100), a next-generation antifibrotic therapy, demonstrated a ...

The consequences of cystic fibrosis (CF) are extensive and affect almost every organ system. The clinical picture is broad and may present with acute or chronic manifestations. Gastrointestinal and ...

The levels of NT-proBNP, left ventricular mass index, maximal wall thickness, and E/E′ ratio were higher in patients with worse neurological stages compared to those in the early neurological stages ...

Dutch obstetrician Monique Haak, MD, demonstrates ultrasound techniques at ISUOG 2025 in Cancún, Mexico. (Photo by Larry Luxner) Congenital heart disease is the most common deformity in fetuses, yet ...

In the pivotal LUNA 3 trial, durable platelet response at week 25 was achieved by 23% of patients with rilzabrutinib compared with 0% with placebo. The US Food and Drug Administration (FDA) has ...

The potential of vitamin D as an add-on therapy lies in its immunomodulatory effect on T cell-mediated and humoral immune responses, complement activation, cytokines, and inflammasomes. Vitamin D ...

In this episode of the Rare Care podcast, Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kfir Oved, PhD, about the latest Israeli research on myasthenia gravis (MG). Dr. Oved ...

Fabry disease treatments consist of pharmacological interventions, nonpharmacological interventions, and surgical procedures. Fabry disease results from a mutation in the galactosidase alpha (GLA) ...

Health regulators in the US are leading a push away from animal models in preclinical studies of potential disease treatments. While animal models are likely to still have a place in translational ...