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), ...

Patients underwent follow-up evaluations on days 4, 10, and 21 post-RS, followed by monthly assessments extending u to 6 months. It is well known that individuals with hemophilia are at an elevated ...

Sandy Siegel, founder and president of the Siegel Rare Neuroimmune Association (Photo by Larry Luxner) Anthropologist Sandy Siegel launched the group after his late wife was diagnosed with transverse ...

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 ...

This case underscores the importance of individualized treatment plans and the need for meticulous perioperative care in patients with bleeding disorders. Among individuals with hemophilia, ...

There were no serious adverse events in children who underwent sessions of robot-assisted gait therapy with the device. ATLAS 2030, the medical lower-body exoskeleton for children is safe for patients ...

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 ...

Although nerve parameters examined in AL amyloidosis patients decreased over the last 24 months, they remained in stage I PND. Patients with hereditary transthyretin amyloid polyneuropathy (ATTR-PN) ...

Living with Pompe disease is a constant battle requiring physical resilience and emotional strength. And it requires the ability to adapt to a life that progressively becomes more challenging. Yet, ...

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 ...

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 ...

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 ...