Case Study: Secukinumab Therapy Improved GPP Flare During Pregnancy
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Rare Disease Researchers Warn NIH Budget Cuts Could Threaten Progress and Endanger LivesWith a $48 billion annual budget, the US National Institutes of Health is the world’s largest public funder of biomedical research. -
Clinicians Treating HAE Have Many Options, With More on the WayFour long-term prophylaxis and 4 on-demand therapies for hereditary angioedema are now on the market. -
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.
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Spring With CAD Brings Unique ChallengesIt's important for people with cold agglutinin disease (CAD) to protect themselves from various types of cold when seasons change.April 2, 2025 -
Spare Me the Toxic PositivityWhen you have a rare disease, like CIDP, you spend a lot of time telling people bad news.April 2, 2025
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On My Way to the 2025 International PBC SummitReceiving top-quality care should be the standard for every patient with primary biliary cholangitis (PBC).April 1, 2025 -
Hope and Struggle With Duchenne Muscular DystrophyA mere 5 months ago, our world turned upside down when our grandson William was diagnosed with Duchenne muscular dystrophy (DMD).March 31, 2025
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How Systemic Mastocytosis Affects Patient Health and Well-BeingStudies indicate that the disease burden of SM is substantial, adversely impacting physical and mental function, alongside employment. -
Improving the Understanding of GPP Epidemiology and Clinical ManifestationsEfforts are underway to better characterize the epidemiology and clinical manifestations of generalized pustular psoriasis (GPP). -
Prioritizing Patient Voices, Experiences in Chronic, Rare DiseasesBy giving our patients a voice, we position ourselves to hear firsthand accounts of the personal impact of chronic, incurable diseases. -
Current, Emerging Biomarkers and Treatments Identified for ATTR-PN and ATTR-CMThis article discusses current and emerging biomarkers and therapeutic strategies for transthyretin amyloid polyneuropathy (ATTR-PN). -
Treating SM-Associated Osteoporosis: A Multidisciplinary ApproachA 23-year-old man with lower back pain and mobility issues was diagnosed with osteoporosis associated with SM and treated for both.
- 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
- Thymidine Kinase 2 Deficiency
- Transthyretin Amyloid Polyneuropathy
- Transthyretin-Mediated Amyloid Cardiomyopathy
- Wilson Disease
