Improved early diagnosis may mitigate necrotizing enterocolitis-related complications such as fetal and neonatal alloimmune thrombocytopenia. Artificial intelligence and machine learning significantly ...

Lisa Butler, president and CEO of the GBS/CIDP Foundation International (Photo courtesy of GBS/CIDP Foundation International) The GBS/CIDP Foundation International aims to provide support, with an ...

(L-R) Christina Hartman, Kathryn Alexander, and Lesa Brackbill discuss newborn screening at the 2025 World Orphan Drug Congress USA in Boston, Massachusetts (Photo by Larry Luxner) Newborn screening ...

Patients with demyelinating polyneuropathy had increased nerve shear wave velocity compared to the controls. Neuropathy is associated with changes in peripheral nerve elasticity, found a new study by ...

The findings showed that 12 out of 19 patients with typical CIDP presented with an incomplete phenotype. Around a third of patients with typical chronic inflammatory demyelinating polyneuropathy (CIDP ...

IN013 is an investigational mRNA therapy aiming to restore ATP7B function and mitigate copper toxicity. The investigational mRNA therapeutic IN013 received both Rare Pediatric Disease Designation and ...

Which of the statements below about the epidemiology of hemophilia are correct? Select one of the options for each question, then submit your answers to see how well you scored and how other test ...

Additive genetic factors account for ~47% of baseline FVIII inhibitor heritability, surpassing F8 mutation contributions. The NOS2A gene encodes inducible nitric oxide synthase, a mediator of innate ...

Life with alpha-1 antitrypsin deficiency AATD) has generally not been easy for me. I have written several columns about trying to communicate complicated things to others (such as family members, ...

Coexisting ATM and del(11q) aberrations were more common in unmutated IGVH CLL. These results support a refinement of current prognostic frameworks in CLL by distinguishing the clinical relevance of ...

When I was in the hospital receiving treatment for chronic inflammatory demyelinating polyneuropathy (CIDP), I was often classified as a “falls risk. That was accurate, given my numb legs and all.

No treatment-related serious adverse events occurred during either study, and all related treatment-emergent adverse events resolved. DTX101 failed to achieve sustained or clinically meaningful ...