Locoregional Therapies Improve Survival in HCC With Portal Vein Thrombosis
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Pioneering Case Illustrates the Promise of Personalized Gene Editing for Rare DiseasesKJ Muldoon has been achieving developmental milestones, but his long-term prognosis is unknown. -
Vasculitis Awareness Month Highlights Advances in Treatment and Ongoing ObstaclesThe nonprofit Vasculitis Foundation advocates for patients with 1 or more of the 20-plus subtypes of the disease. -
A Kansas Man’s Lifelong Struggle With Limb-Girdle Muscular DystrophyDutch filmmaker Daimon Xanthopoulos recently produced a 25-minute documentary about Donavon Decker’s life and relationships. -
Regional Pockets of HAE Persist Due to Founder Effect, Affecting Generations of FamiliesHAE affects approximately 1 in 50,000 people globally, but is far more prevalent in a few select geographic regions. -
New Prader-Willi Therapy Gives Parents Rare Hope for Managing HyperphagiaDiazoxide choline is believed to activate the ATP-sensitive potassium channel, which may decrease secretion of peptides that regulate appetite.
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How I Manage Patient BurnoutLiving with LGMD, there have been plenty of times when I’ve had to deal with healthcare workers not taking my health seriously.June 12, 2025 -
Hope Rising in the MG CommunityBefore I was diagnosed in July 2019 with myasthenia gravis (MG), I had never heard of the disease.June 11, 2025
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Living With Pompe Disease: The Unseen Battle Against BurnoutI am sitting here reflecting on my journey with Pompe disease, and I'm reminded of the challenges that come with living with a rare genetic disorder.June 11, 2025 -
When Living With Pompe Reaches a Breaking PointRecently, I had my infusion scheduled as I do every other week to manage Pompe disease.June 11, 2025
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Ensuring the Optimal Management of Myelofibrosis-Associated AnemiaThe burden of care for myelofibrosis-associated anemia is high, especially among patients who are transfusion-dependent. -
The Role of Inflammation as a Key Driver in FNAITA recent study indicated that placental inflammation may be a key disease modulator in fetal and neonatal alloimmune thrombocytopenia. -
When Is Genetic Testing Appropriate to Investigate a Diagnosis of HAE in Children?A case study demonstrates how genetic testing can be useful in children with a positive family history of hereditary angioedema. -
Enhancing Palliative Care in HCCPalliative care should be integrated into the early treatment and management of patients with hepatocellular carcinoma (HCC). -
AAV Pathophysiology: Rare Inborn Errors of ImmunityAlthough there are early data, more research is needed regarding why ANCA develop in an inborn error of immunity and whether they play a role in AAV.
- 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
- Fabry Disease
- Fetal and Neonatal Alloimmune Thrombocytopenia
- Friedreich Ataxia
- Gastrointestinal Stromal Tumor
- Gaucher Disease
- 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
