Rare Disease Advisor

China Approves Subcutaneous Batoclimab for AChR-Positive Generalized Myasthenia Gravis

Based on phase 3 trial results, China’s NMPA has approved subcutaneous batoclimab for adults with AChR-positive myasthenia gravis (MG).
Rare Disease Advisor

Modernizing Global Data Exchange: Reagan-Udall Foundation to Host Public Meeting

The Reagan-Udall Foundation will host a public meeting exploring how to modernize global exchange of regulatory data for ...
Rare Disease Advisor

New Score May Identify Patients With Pancreatitis at High Risk for Venous Thromboembolism

A 6-item score may help identify an increased risk for thrombosis among hospitalized patients with pancreatitis, a ...
Rare Disease Advisor

Olfactory Decline Distinguishes GBA1-Associated Parkinson Disease From Gaucher Disease

Individuals with GBA1-associated Parkinson disease may experience faster rates of olfactory decline than those with Gaucher ...
Rare Disease Advisor

Therapy-Related MDS Carries Poor Survival After Gynecologic Cancer Treatment

Three patients in a 5-case series of therapy-related myelodysplastic syndromes (t-MDS) after gynecologic cancer treatment ...
Rare Disease Advisor

Researchers Call for Exploring Galectin-9 as Preeclampsia Target in Systemic Mastocytosis

Researchers proposed testing galectin-9 as an add-on to low-dose aspirin or to standard preeclampsia treatment during pregnancy in SM.
Rare Disease Advisor

Misdiagnosis and Delayed Diagnosis Shown to Be Common in ILD

Misdiagnosis and late diagnosis are highly common in patients with interstitial lung disease (ILD), according to a new study.
Rare Disease Advisor

Acalabrutinib Improves Progression-Free Survival in High-Risk Mantle Cell Lymphoma

Acalabrutinib plus bendamustine-rituximab improved progression free survival compared with bendamustine-rituximab alone in ...
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Inflammatory Markers Identify Patients With MDS at Higher Risk of Mortality

Elevated C-reactive protein and absolute monocyte count were associated with poorer overall survival among patients with MDS.
Rare Disease Advisor

CD71 Levels Reveal Distinct Erythroid Defects in MDS-RS

Patients with MDS with ring sideroblasts had lower CD71 expression and higher proerythroblast proportions than those with ...
Rare Disease Advisor

Post-transplant PNI Changes Correlate With Engraftment and ICU Risk

In a mixed-diagnosis ASCT cohort that included patients with MCL, baseline PNI did not predict early transplant-related ...
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Myasthenia Gravis

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 ...