Assessing Epidemiological Data Points The findings from this study echo that of another by Trivella and colleagues, also published in Hepatology Communications. This 2023 study is a literature review ...

A single intravenous infusion of the investigational adeno-associated virus (AAV) gene therapy scAAV2/8-LP1-hFIXco demonstrated durable clinical benefit and long-term safety in patients with severe ...

Combining handcrafted and automatic features within a dual-feature framework enhances the diagnostic accuracy for Philadelphia chromosome-negative myeloproliferative neoplasms, including myelofibrosis ...

Among patients with severe hemophilia A and B, treatment with the monoclonal antibody marstacimab has been associated with reduced rates of bleeding, according to a systemic review and meta-analysis ...

In a letter to the editor of the Indian Journal of Radiology and Imaging, Yadav and colleagues concluded that the Pettersson Score remains valuable to assess radiographic severity in children. This is ...

Patients with myasthenia gravis (MG) have a changed CD4 T cell phenotype, according to a new study published in the Journal of Neurochemistry. The authors of the study suggested that the frequency of ...

Adult patients with Fabry disease have a lower lean mass compared to healthy people, according to a new study published in the Journal of Rare Diseases. The accumulation of lyso-Gb3 is also correlated ...

Neurodevelopmental and mental disorders may be common in boys and adults with Duchenne muscular dystrophy (DMD), according to a study published in the Archives of Disease in Childhood. Over a third of ...

Patients with Fabry disease appear to have a higher incidence of renal cell carcinoma than the general population, according to a recently published study in Molecular Genetics and Metabolism. “The ...

“Andembry, a novel once-monthly subcutaneous treatment that inhibits factor XIIa, is a welcome addition to the HAE treatment landscape,” said Anthony J. Castaldo, CEO and chairman of the board of the ...

The US Food and Drug Administration (FDA) has granted a Priority Review designation to a New Drug Application (NDA) for TransCon ® CNP (navepegritide) for the treatment of children with achondroplasia ...

Higher rates of all types of hereditary angioedema (HAE) in the United States have been observed in a claims-based study published in the Annals of Allergy, Asthma, and Immunology. An analysis of ...