Vigilance and Targeted Therapy Lead to Better Prognosis in CNS-Involved CLL/SLL
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California Charity Helps Minorities, Undocumented Patients Access Vital Hemophilia TreatmentThe Hemophilia Foundation of Southern California serves 3800 members, at least half of whom are native Spanish speakers.
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Global Liver Institute’s New Head of Rare Diseases Is Tennessee Woman With AATDA mysterious illness plagued Kristin Hatcher’s family for 4 generations, until her father was diagnosed with AATD.
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Patients With Rare Diseases Struggle in Aftermath of Los Angeles WildfiresThe impact of the deadly Los Angeles County wildfires on rare disease patients is especially painful.
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Stem Cell Therapies Eyed as a Potential Cure for NMOSDOne path to curing NMOSD may lie with stem cell therapy, several neurologists who spoke at a recent conference said.
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The Challenges Clinicians Face in Detecting and Treating FNAITRare antigens, ill-defined guidelines, and shortcomings in screening are among the challenges clinicians face in diagnosing and treating FNAIT.
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Earning a Living and the Burdens of MGWith generalized myasthenia gravis (MG), our brains are still functioning.February 11, 2025
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Knowing When Your Medication Is No Longer WorkingRight after a diagnosis of Pompe disease, you are instructed to start treatment, which is a biweekly infusion of enzyme replacements.February 11, 2025
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Learning the Keys to Staying Strong While Managing ITPCoping, hope and resilience can help people with immune thrombocytopenia (ITP) handle the struggles of managing the condition.February 6, 2025
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Apps That Work for Me and My AATDColumnist with alpha-1 antitrypsin deficiency (AATD) explores apps that may be helpful for managing symptoms of the condition.February 6, 2025
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Splenic Artery Aneurysms Possibly Associated With IgG4-RDMore research is needed to determine the connection between IgG4-RD and splenic artery aneurysms.
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Managing Concurrent HDFN and FNAIT: A Rare CaseA neonate with known HDFN was discovered to also have FNAIT after birth, necessitating immediate, intensive intervention.
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Assessing the Behavioral and Mental Aspects of Huntington DiseaseHuntington disease is most often characterized by a triad of motor, behavioral, and cognitive dysfunction, and here's how clinicians are managing it.
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Avoiding Misdiagnosis in Cases of AAV Initially Resembling MalignancyGranulomatosis with polyangiitis, a form of AAV, can sometimes resemble malignancy, leading to occasional instances of misdiagnoses.
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Supporting Health-Related Quality of Life in Indolent Systemic MastocytosisResearch into how the health-related quality of life of patients with indolent systemic mastocytosis is affected is vital to disease management.
- 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
- 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
- Transthyretin Amyloid Polyneuropathy
- Transthyretin-Mediated Amyloid Cardiomyopathy
- Wilson Disease