Claseprubart incorporates YTE half-life extension technology, which is designed to enable infrequent subcutaneous self-administration. A phase 3 study is underway to evaluate the investigational ...

As of May 2025, 62 active clinical trials are ongoing for Fabry disease, including 25 interventional and 37 observational studies, of which 8 are patient registries. Among the trials with known ...

The antibodies were slightly more common in double seronegative MG, at 3.9%, than in seropositive MG, at 1%. In patients with suspected myasthenia gravis (MG), LRP4-IgG antibodies are rare, with poor ...

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

Outcomes improved in patients who received nomlabofusp, while scores worsened in a matched reference cohort from the FACOMS natural history database. Larimar Therapeutics announced that the US Food ...

Patient satisfaction points to the need for novel CIDP-targeted treatments. Misdiagnoses, diagnostic delays, and persistent disease burden despite adequate treatment remain key challenges in chronic ...

Derived thresholds of 4.5 kg for deterioration, 5 kg for improvement, and 20% for either deterioration or improvement are significantly more specific and accurate. More stringent thresholds than what ...

Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...

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 ...

The highest in-hospital mortality and complication rates were found among Asian or Pacific Islander patients, and the longest hospital stays were found among African Americans and those classified as ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

The study reported no serious adverse events in either the guanfacine extended-release or placebo groups. Guanfacine extended-release (GXR) shows promise for managing behavioral symptoms in ...