At 18 months, progression-free survival was 96%, and overall survival was 96.8%, with no patients requiring subsequent therapy. The measurable residual disease (MRD)-guided combination of zanubrutinib ...

Although the 3 mental health measures (anxiety, depression, and anger) improved, they increased less than the physical and social scores. Many aspects of social and physical health improve in patients ...

Neurological improvement was reported throughout the trial, suggesting continued improvements may still be reported for an extended time period. In patients with Wilson disease (WD), long-term ...

Pediatric-onset of GPA is a rare occurrence, with an incidence rate of the disease at 1.8 per 1,000,000 population in US children aged 18 years or younger. In patients with pediatric-onset ...

Overall, 42.9% (9 of 21) of the participants reported experiencing GPP-associated symptoms on a daily basis. Many patients with generalized pustular psoriasis (GPP) report experiencing chronic ...

(Getty Images) Six patients with impaired LVEF at baseline experienced more than 10% improvement. CHICAGO—Researchers detailed the histological, cellular, and molecular effects of tafamidis therapy in ...

Credit: Getty Images Moderately strong correlations were identified between global disease burden and both ventricular mass index and extracellular volume. CHICAGO—Quantitative uptake of ...

Another patient who has alpha-1 antitrypsin deficiency (AATD) recently spoke up in our support group. She reports that many people do not believe her when she says she has this condition. I can relate ...

The study aims to measure changes in symptoms and disease activity scores as well as treatment-emergent adverse events. A Chinese study is recruiting patients with relapsed or refractory autoimmune ...

Everyone with Pompe disease is diagnosed at different times in life, so the level of muscle weakness differs from person to person. My daily challenges might be different from those of the next person ...

Future studies are needed to confirm darbepoetin alfa's efficacy in reducing neurodevelopmental issues that arise in some neonates requiring transfusions. Neonates who received intrauterine ...

Patients with MPO-ANCA+ spinal hypertrophic pachymeningitis had worsening spinal symptoms, lesions in the dura matter, more serious neurological symptoms, and a frequent need for emergency surgery.