Mucin 3A Regulates Proliferation and Metastasis of Cholangiocarcinoma Cells
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IgG4-RD: A Mimicker of Multiple MalignanciesIn the past few years alone, researchers have reported myriad cases of IgG4-RD being mistaken for some type of malignancy.
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Boston Dermatologist Says the Most Severe Form of Psoriasis Can Be a ‘True Emergency’Generalized pustular psoriasis is the most serious form of psoriasis, with more than half of all patients with the condition needing hospitalization.
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Lifelong Care of the Patient With AchondroplasiaA polysomnography is recommended in the first year of life for patients with achondroplasia to identify abnormalities in breathing patterns.
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Rare Disease Researchers Warn NIH Budget Cuts Could Threaten Progress and Endanger LivesWith a $48 billion annual budget, the US National Institutes of Health is the world’s largest public funder of biomedical research.
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Clinicians Treating HAE Have Many Options, With More on the WayFour long-term prophylaxis and 4 on-demand therapies for hereditary angioedema are now on the market.
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The Consequences of Temperature Swings When Coping With CADTemperature variations can cause hemoglobin fluctuations in people with cold agglutinin disease (CAD), leading to hemolysis and other symptoms of disease.April 16, 2025
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Living With Hemophilia: A Journey of Adaptation and AwarenessMy journey has taught me that while hemophilia may be a part of my life, it does not define me.April 15, 2025
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A Mismatched Medication Creates Choppy Seas for This Traveler With MSWith a little planning, people with multiple sclerosis (MS) can enjoy traveling on cruise liners with helpful accessibility options.April 14, 2025
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Remember Your Emotional Well-Being When Caring for Your ITPFor patients with immune thrombocytopenia (ITP), it is important to care for one's emotional well-being along with physical health.April 14, 2025
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C5 Polymorphisms and Their Impact on Eculizumab EfficacyResearchers have investigated the C5 polymorphisms that impact the response to eculizumab in patients with PNH.
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Early Diagnosis of ATTR-PN May Be Both Vital and AchievableThe early diagnosis of transthyretin amyloid polyneuropathy is both vital to secure long-term outcomes and achievable.
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Massive Hemoptysis in IgG4-Related Respiratory DiseaseA patient with recurrent hemoptysis was eventually diagnosed with IgG4-related respiratory disease following extensive investigations.
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Using Markers of Prognosis to Estimate Survival in ATTR-CMThe ability to accurately predict survival/life expectancy in ATTR-CM can better inform clinical decision-making.
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Novel Imaging Tools May Be Used to Monitor Therapeutic Changes in ATTR-CM and ATTR-PNThis article highlights the potential of 99mTc-PYP SPECT/CT in monitoring therapeutic responses to siRNA treatment in ATTR.

- Achondroplasia
- Acute Lymphoblastic Leukemia
- Alagille Syndrome
- Alpha-1 Antitrypsin Deficiency
- ANCA-Associated Vasculitis
- Cholangiocarcinoma
- Chronic Inflammatory Demyelinating Polyneuropathy
- Chronic Lymphocytic Leukemia
- Cold Agglutinin Disease
- Cystic Fibrosis
- Diffuse Large B-Cell Lymphoma
- Dravet Syndrome
- Duchenne Muscular Dystrophy
- Fetal and Neonatal Alloimmune Thrombocytopenia
- Friedreich Ataxia
- Gastrointestinal Stromal Tumor
- Generalized Pustular Psoriasis
- Hemolytic Disease of the Fetus and Newborn
- Hemophilia
- Hepatocellular Carcinoma
- Hereditary Angioedema
- Huntington Disease
- Idiopathic Pulmonary Fibrosis
- IgG4-Related Disease
- Immune Thrombocytopenia
- Lennox-Gastaut Syndrome
- Long Chain Fatty Acid Oxidation Disorder
- Lysosomal Acid Lipase Deficiency
- Medullary Thyroid Carcinoma
- Multiple Sclerosis
- Myasthenia Gravis
- Myelodysplastic Syndromes
- Myelofibrosis
- Neuromyelitis Optica Spectrum Disorder
- Paroxysmal Nocturnal Hemoglobinuria
- Pompe Disease
- Prader-Willi Syndrome
- Primary Biliary Cholangitis
- Primary Central Nervous System Lymphoma
- Pulmonary Arterial Hypertension
- Sickle Cell Disease
- Spinal Muscular Atrophy
- Systemic Mastocytosis
- Systemic Sclerosis
- Thymidine Kinase 2 Deficiency
- Transthyretin Amyloid Polyneuropathy
- Transthyretin-Mediated Amyloid Cardiomyopathy
- Wilson Disease