Patients With NMOSD May Benefit From Rituximab and Mycophenolate Mofetil
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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. -
GBS/CIDP Foundation Helps Promote Awareness of Rare Debilitating NeuropathyThe GBS/CIDP Foundation International aims to provide support, with an emphasis on patients and caregivers meeting each other. -
Advocates Seek Solutions After HHS Ends Newborn Screening Advisory PanelNewborn screening in the US currently identifies about 14,000 babies with serious conditions annually.
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Discovering How Word Puzzles, Games Help Improve Some MS SymptomsOnline games can be important to someone with multiple sclerosis (MS), where cognitive fog and fatigue are both common symptoms.May 15, 2025 -
How Other People With AATD Inspire MeKristin Hatcher, a patient with AATD and head of The Global Liver Institute, inspires me. Her story reveals the many issues patients face.May 14, 2025
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Steroids: The Good, the Bad, the Ugly for ITPPatients with a relapse of immune thrombocytopenia (ITP) are often prescribed steroid treatment, which can come with difficult side effects.May 9, 2025 -
Is There a Connection Linking AATD and Atrial Fibrillation?Knowing a potential connection between alpha-1 antitrypsin deficiency (AATD) and atrial fibrillation helps patients be aware of complications.May 9, 2025
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Quantifying Disease Burden in PAHThe disease burden in PAH is both personal and macroeconomic; both have to be approached with strategy, tact, and empathy. -
Patients With PBC Could Present With Hypoglycemic Coma EpisodesRecurrent hypoglycemic coma episodes may lead to a primary biliary cholangitis diagnosis. -
Early Diagnosis May Be Useful in ATTR-CM CareThe early diagnosis of ATTR-CM allows for early interventions to be initiated that may strengthen long-term outcomes. -
Rare CNS Involvement Identified in Infantile-Onset TK2dResearch highlights rare CNS involvement in cases of infantile-onset thymidine kinase 2 deficiency (TK2d). -
The Importance of Antenatal Care in Pregnancies at Risk of FNAITBasic, adequate antenatal care is vital in pregnancies at risk of fetal and neonatal alloimmune thrombocytopenia.
- 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
