Diagnosis by 2 HCPs Over 2 Quarters May Offer Reliable GPP Prevalence
-
National Amyloidosis Awareness Month: Expert Says Future of Treatment Is BrightAmyloidosis, which has several types, is diagnosed in at least 4000 Americans per year. -
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. -
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. -
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. -
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.
-
How Cold Agglutinin Disease and UTIs OverlapWhen you have a rare disease such as cold agglutinin disease (CAD), you need to be diligent to avoid infections.March 12, 2025 -
How My Liver Biopsy Turned Into a NightmareI do not want to discourage other primary biliary cholangitis (PBC) patients from using this procedure.March 11, 2025
-
Encouraging Others to Learn About ITP, My Rare DiseaseWhen diagnosed with a rare disease like immune thrombocytopenia (ITP), it's important to find ways to share your condition and experience.March 7, 2025 -
William’s Journey: Tiptoeing Through the Long Road to a DMD DiagnosisDuchenne muscular dystrophy (DMD) was not something that crossed our minds at first, and it certainly wasn’t something doctors suggested early on.March 7, 2025
-
Momelotinib May Be a Promising Treatment Option for Patients With Myelofibrosis-Associated AnemiaMyelofibrosis-associated anemia can often result in significant levels of fatigue, impairing health-related quality of life. -
Patients With ATTR-CM May Benefit From Early Screening and DiagnosisOne of the most effective ways of improving outcomes is the implementation of early screening and diagnosis of ATTR-CM. -
What Is the Relationship Between IgG4-RD and Autoimmune Disease?Research suggests there is some relationship between IgG4-RD and autoimmune rheumatic diseases that deserves further exploration. -
Current and Emerging Strategies for Managing AchondroplasiaThe management of achondroplasia involves several strategies, including the first approved precision medicine, vosoritide. -
Using Epidemiology to Capture Vital Data on NMOSDEpidemiological data on rare diseases such as NMOSD helps inform public health decision-making and shapes future studies.
- 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
