Increasingly, modern medicine is making room for patients to better express themselves through self-reported outcomes, especially for rare diseases like SM. There is an old adage in medicine: ...

As president and CEO of the GBS/CIDP Foundation International, Lisa Butler oversees a staff of 20 and an annual budget of approximately $6 million. Larry Luxner, senior correspondent for Rare Disease ...

The PBC-10 is considered useful for the rapid screening of patients in busy outpatient clinical settings who may have severe subjective symptoms and impaired HRQOL. The Japanese version of the Primary ...

Antifibrotic treatment led to significant alterations in the concentrations of octanal, 3-methylfuran, and m-cymene. Specific profiles of volatile organic compounds (VOCs) in the exhaled breath of ...

In this study, approximately 90% of the pregnant women with hematologic disorders who experienced maternal near-miss and mortality had thrombocytopenia. Among pregnant women, maternal near-misses and ...

Among participants who attained PFS at 24 months, the 5-year overall survival rate was 96.7%. Researchers obtained data from 146 patients with newly diagnosed, transplant-eligible PCNSL who had ...

Patients undergoing deformity correction experienced longer external fixation and consolidation periods, likely due to older age and more complex biomechanics. Simultaneous multiplanar correction of ...

Vidofludimus calcium reduced the relative risk of disease worsening events in patients without gadolinium-enhancing lesions at baseline by 29% compared to placebo. Immunic Therapeutics announced ...

Multivariable analysis showed that patients with primary MF with an absolute basophil count above 0.8 × 109/L had worse overall survival. Basophilia is closely associated with high-risk primary ...

With the right therapeutic strategies and patient adherence, the risk of breakthrough hemolysis in PNH can be minimized. Treatment optimization and medical education are key factors in preventing ...

The 3-year overall survival rates of patients undergoing hematopoietic stem cell transplantation from a mismatched donor improved in recent years. Survival rates post-hematopoietic stem cell ...

The authors speculated the increased risk could be due to mutations of driver genes that specifically impact megakaryopoiesis and platelet formation. Individuals with clonal hematopoiesis of ...