Improvements in each measure were greater with eplontersen compared to placebo than the estimates of meaningful differences. Eplontersen treatment has a clinically meaningful effect on neuropathic ...

Results showed a significantly higher prevalence of liver disease among patients with chronic pruritus compared to healthy controls. Patients with liver diseases of different etiologies, including ...

Sotatercept at 0.7 mg/kg subcutaneous every 3 weeks optimally balanced efficacy and safety for those with PAH. Sotatercept, a practice-changing therapy for pulmonary arterial hypertension (PAH), ...

The total score obtained by patients with FA in the Mild Behavioral Impairment Checklist (MBI-C) was significantly higher than that obtained by healthy controls. Neuropsychiatric symptoms, especially ...

Asian-type DEL prevalence among Malay individuals was 25% in donors and 21% in patients, exceeding East Asian averages. Accurate knowledge of blood group variants, including the Asian-type DEL, is ...

Rosalind Kalb is lead senior programs consultant for Can-Do MS, a Colorado-based nonprofit health and wellness organization. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

Patients who had bone fractures had a 1.5 times higher decline of total body bone mineral density Z-score per year and a worse adiposity profile than those with no fractures. Fragility fractures are ...

Test Your Knowledge About the Etiology of Cold Agglutinin Disease ...

The mortality rates were 15.5% for the ibrutinib group and 16.3% for the bendamustine group. Significant cardiovascular risks are associated with ibrutinib treatment in patients with chronic ...

As of November 2024, approximately 156 clinical trials for Duchenne muscular dystrophy (DMD) are ongoing or pending: 16 trials not yet recruiting, 65 currently recruiting, 28 trials that are active ...

After an open-label phase 2 trial found proof-of-concept efficacy for nipocalimab in the treatment of pregnant women at high risk of hemolytic disease of the fetus and newborn (HDFN), researchers are ...