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 ...

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 ...

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 ...

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 ...

As of November 2024, approximately 137 clinical trials for spinal muscular atrophy (SMA) are ongoing or pending: 8 trials that are not yet recruiting, 74 trials that are currently recruiting, 26 ...

For T2 signal intensity (SI), there was a significant and strong correlation between SI and the number of days since the last IUT, with SI initially reduced and recovering over time. A recent study ...

This particular combination of mutations and comorbidities led to an aggressive disease course, which was refractory to standard treatments. The report, published in Case Reports in Hematology, ...

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 ...

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 ...

Steve Van Wormer is cofounder and director of the Phaware Global Association, a nonprofit group that raises awareness of pulmonary hypertension (Photo by Larry Luxner) I’m Aware That I’m Rare has had ...