Rare Disease Advisor

RareDiseaseAdvisor.com stands out as the leading online platform for news, insights, and resources aimed at healthcare providers treating rare diseases. This site focuses on filling the gaps in information for medical professionals who are looking for up-to-date knowledge on the rare conditions they encounter, offering original, reliable, and engaging content.

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Resection Margin May Not Influence OS in Patients With pCCA and LN Metastasis

1 day ago | rarediseaseadvisor.com | Juliette Siegfried

The overall survival (OS) of patients with perihilar cholangiocarcinoma (pCCA) and lymph node metastasis is not affected by surgical margin status, according to results published in the European Journal of Medical Research. An analysis of LN metastasis-positive patients who had curative resection for pCCA showed a comparable 5-year OS between those with and without a positive surgical margin.
Successful Outpatient TKA in a Patient With CAD: A Case Report

1 day ago | rarediseaseadvisor.com | Juliette Siegfried

With multidisciplinary collaboration and coordination, outpatient total knee arthroplasty (TKA) can be performed in patients with cold agglutinin disease (CAD), according to a case report published in the Canadian Journal of Anesthesia. The report emphasized the need for appropriate intraoperative precautions, postoperative instructions, and careful monitoring to ensure an optimal outcome.
Role of Antibody Classes in IgG4-RD Helps Reveal Potential Disease Targets

1 day ago | rarediseaseadvisor.com | Sheila Jacobs

In those with immunoglobulin G4-related disease (IgG4-RD), antibodies from a variety of classes play key roles in the underlying immunologic mechanisms of the disorder and in disease progression, according to findings from a review published in Rheumatology & Autoimmunity. The precise immunopathology involved in the development of IgG4-RD remains unclear.
Patients at MGFA Conference Offer Insights on Living With Myasthenia Gravis

2 days ago | rarediseaseadvisor.com | Larry Luxner

THE HAGUE, Netherlands—Four women from four European countries shared what it’s like to live with myasthenia gravis (MG), during a session at Myasthenia Gravis Foundation of America (MGFA)’s first-ever gathering outside the US. The MGFA 15th International Conference on Myasthenia and Related Disorders attracted 650 clinicians, researchers and patient advocates from 44 countries to this Dutch city.
Central Adrenal Insufficiency Is a Rare, Life-Threatening Manifestation of PWS

2 days ago | rarediseaseadvisor.com | Juliette Siegfried

Central adrenal insufficiency (CAI) is a severe and potentially life-threatening endocrinological manifestation of Prader-Willi syndrome (PWS), according to results presented at the 2025 Joint Congress of ESPE and ESE. An analysis of adrenocorticotropic hormone (ACTH) and morning plasma cortisol (MPC) levels among patients with PWS showed that MPC monitoring may be useful as an initial screening step for CAI.
A Kansas Man's Lifelong Struggle With Limb-Girdle Muscular Dystrophy

2 days ago | rarediseaseadvisor.com | Larry Luxner

DALLAS—In 1999, Donavon Decker—a 37-year-old air traffic controller from Huron, South Dakota—made medical history when he became the world’s first recipient of gene therapy for a neuromuscular disease. Decker, now 62 and living just outside Wichita, Kansas, says the groundbreaking procedure ultimately changed the trajectory for a whole range of other diseases.
Addressing the Muti-Faceted Burden of Achondroplasia

3 days ago | rarediseaseadvisor.com | Ryner Lai

Achondroplasia is an incurable, lifelong condition that is primarily characterized by dwarfism, spinal abnormalities, and mobility issues. A diagnosis can be made as early as the second or third trimester of pregnancy due to the genetic nature of this disorder. Whether it is discovered neonatally or postnatally, the diagnosis can be a complex, emotional discovery. Fortunately, the art of breaking challenging news to a patient is one that medical students are specifically trained in doing.
Novel ML-Based MRI Radiomics Model Differentiates PCNSL From GB and BM

3 days ago | rarediseaseadvisor.com | Juliette Siegfried

A new magnetic resonance imaging (MRI)-based radiomics and machine learning (ML) model can accurately identify primary central nervous system lymphoma (PCNSL) and distinguish it from intracranial glioblastoma (GB) and brain metastases (BM), according to a new study published in Frontiers in Oncology. The innovative MRI-based artificial intelligence (AI) approach can preoperatively diagnose brain lesions, which is essential for determining the optimal treatment strategy.
MS Linked to Less Cardiovascular Autonomic Dysfunction Than NMOSD

3 days ago | rarediseaseadvisor.com | Ozge Ozkaya

Thai patients with neuromyelitis optica spectrum disorder (NMOSD) show a significantly higher prevalence of cardiovascular autonomic dysfunction than those with multiple sclerosis (MS), at 95% vs 55%, respectively, according to a new study published in the journal Multiple Sclerosis and Related Disorders.
Patients With MF May Benefit From Pulmonary Hypertension Monitoring

3 days ago | rarediseaseadvisor.com | Ozge Ozkaya

Patients with primary myelofibrosis (MF) have the highest risk of pulmonary hypertension among patients with Philadelphia chromosome-negative myeloproliferative neoplasms (Ph-MPNs), found a new study published in the journal Annals of Hematology. Advanced age and high risk of pulmonary hypertension are independent risk factors for patient survival, the study also found.