Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

Upper-to-lower body segment ratio decreased significantly from baseline relative to untreated comparators in both age groups. Continuous treatment with vosoritide in children with achondroplasia shows ...

In a cross-sectional study, the researchers reported psychiatric comorbidities in 46.5% of 43,270 adults with neurofibromatosis. The most common psychiatric comorbidities included mood disorders (22.1 ...

Genotyping may inform individualized methotrexate dosing strategies to mitigate adverse events. These results support the polymorphism’s potential utility as a pharmacogenetic biomarker to guide ...

Consolidation or maintenance therapy significantly improved progression-free survival and overall survival. Zanubrutinib, lenalidomide, temozolomide, and rituximab (RLZT) combined with or without high ...

Prompt discontinuation of the suspected drug is critical for recovery. Drug-induced immune hemolytic anemia (DIIHA) should be investigated in suspected cases of warm autoimmune hemolytic anemia (warm ...

Wild-type ATTR-CM comprised 76% of cardiac amyloidosis cases, with prevalence highest in Asia and lowest in North America. Cardiac amyloidosis (CA) is present in approximately 10% of patients with ...

Notably, the CRL did not identify safety or efficacy concerns. Aquestive Therapeutics, Inc. announced that the US Food and Drug Administration (FDA) has issued a complete response letter (CRL) for ...

The authors argue that the long-held label of "uniformly lethal" may no longer be appropriate and can inadvertently bias clinical decision-making. Homozygous achondroplasia is traditionally ...

Tafamidis clinically stabilized neuropathy in most patients who developed acquired transthyretin amyloidosis following a domino liver transplantation. Patients who receive livers from individuals with ...

Spesolimab is a humanized monoclonal immunoglobulin G1 antibody and is the first therapy to target the IL-36 pathway, a key driver of GPP disease processes. Spesolimab has an acceptable safety profile ...

Although therapeutic advancements have offered better symptom control in GPA, it is still associated with a high risk of relapse. Organ involvement upon diagnosis in granulomatosis with polyangiitis ...