Real-World EGPA Treatment in Japan Diverges From Guidelines
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Pharma Companies Race to Develop New Therapies for Older Patients With SMAIntrathecal onasemnogene abeparvovec aims to address the genetic root cause of SMA by replacing the SMN1 gene in a single dose.
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MGFA Honors British Neurologist With First Lifetime Award for Groundbreaking MG ResearchAfter starting in clinical medicine at her parents' urging, Dr. Vincent switched to her field of interest: basic research.
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Orthopedic Care for Achondroplasia Rapidly Evolving as New Strategies EmergeOrthopedic management plays a central role in the care of patients with achondroplasia.
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Emerging Solutions Could Make TK2d Easier to Recognize and TreatTK2d has long existed in a therapeutic void, with patients having few, if any, disease-modifying treatment options.
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SMA Continues to Challenge Patients Who Couldn’t Be Treated Early in InfancyLyza Weisman spoke at the recent MDA Clinical & Scientific Conference of no longer being able to do the 'little things' that make a life.
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Making MS Life Easier With In-Home InnovationsInnovative accessibility applications can improve quality of life for patients with multiple sclerosis (MS) who use mobility devices.July 25, 2025
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Interacting With AATD Infusion NursesIndividuals with alpha-1 antitrypsin deficiency (AATD) may be prescribed home AATD infusion therapy, which involves infusion nurses.July 24, 2025
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Deconstructing the Link Between CAD and WAHAIndividuals with cold agglutinin disease (CAD) can also have warm autoimmune hemolytic anemia (WAHA), a condition known as mixed type AIHA.July 23, 2025
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Coping With Hemophilia: Finding Joy and Safety in Hobbies and CreativityIt's important to have hobbies that help you decompress. You can turn to these outlets for relaxation and comfort when you’re feeling stressed.July 22, 2025
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Understanding the Impact of GPP on Quality of Life OutcomesGeneralized pustular psoriasis (GPP) contributes to consistently negative outcomes across various quality of life parameters.
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A Holistic Approach to Improving Quality of Life Among Patients With SCDTo make a real difference in the lives of patients with SCD, there should be a focus on optimizing care across a vast spectrum of concerns.
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Improving the Diagnostic Rate of IgG4-RDWith the characterization of IgG4-RD, this disease can finally be managed in a comprehensive manner—provided that it is correctly diagnosed, of course.
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Cardiac Amyloidosis and Pulmonary Sarcoidosis May CoexistAn elderly patient with a history of pulmonary sarcoidosis was diagnosed with transthyretin amyloid cardiomyopathy.
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Assessing Efforts to Quantify Therapeutic Goals in Myasthenia GravisTo better achieve clinical progress, therapeutic goals in myasthenia gravis must be objectively and unambiguously defined.

- 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
- Limb Girdle Muscular Dystrophy
- 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