Endothelin-1 May Be a Novel Biomarker of Systemic Sclerosis
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National Amyloidosis Awareness Month: Expert Says Future of Treatment Is BrightAmyloidosis, which has several types, is diagnosed in at least 4000 Americans per year.
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More PAH Therapies Than Ever Before, Yet Time to Diagnosis Hasn’t ImprovedThe number of treatments for PAH continues to rise, but it still takes an an average of 3 years from symptom onset to diagnosis.
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Colorado-Based ‘Can Do MS’ Helps People With Multiple Sclerosis Live Healthier LivesCan Do MS provides free programs on topics such as nutrition, exercise, MS symptoms, relationships, and employment.
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Smoking and Air Pollution May Exacerbate MDS and Shorten LifespanBlack carbon and PM2.5, toxins with known links to cancers, correlated highly with MDS disease severity scores in a New York study.
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Rare Disease Doctors Tout Value of Social Media While Warning of Online MisinformationThe average American has 7.1 social media accounts, according to industry statistics.
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Adding a Little Color and Creativity to My Wheels as a Patient With CIDPI was amid my chronic inflammatory demyelinating polyneuropathy (CIDP) treatment and was spending half my life in hospital beds.March 17, 2025
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Coping With Joint Pain While Living With Pompe DiseaseOver the past weeks, I have been experiencing joint pain.March 17, 2025
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My Goals for 2025 as a Person With AATDHaving a framework of rare disease, in this case alpha-1 antitrypsin deficiency (AATD), can change your priorities and life goals.March 14, 2025
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Living With ITP: Knowing Your Body and Its LimitsI found myself in a very uncomfortable and unsettling situation, all because I chose to share about my illness, immune thrombocytopenia (ITP).March 14, 2025
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Aplastic Anemia and Concurrent PNH: Challenges and OpportunitiesAplastic anemia and PNH are both rare disorders, but in some patients they overlap, representing a significant medical challenge.
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Patients With AAV at Risk of Cardiovascular DiseaseStudies indicate that patients with AAV are at significant risk of developing cardiovascular disease, requiring medical vigilance.
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The Gender Difference in CIDP: Why Are Males Affected More?This article explores sex differences in the prevalence and outcomes of CIDP, and underlying factors contributing to the male predominance.
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Overcoming Treatment Inertia in the Use of Newer Immunotherapies for NMOSDA number of newly approved immunotherapies have shown considerable promise in changing the treatment landscape of NMOSD.
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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.

- 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