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) ...

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 ...

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 ...

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, ...

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 ...

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 ...

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 ...

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 ...

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 ...