Donidalorsen Approved in Canada for HAE Prevention
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Hermansky-Pudlak Syndrome: A Multisystem Disease That Often Leads to Pulmonary FibrosisHPS has 11 subtypes; 100% of patients with type 1, 2, or 4 will develop pulmonary fibrosis, usually by their mid-30s. -
For Cancer Care, the AI Revolution Has Already Begun, Dr. Douglas Flora SaysIn his book, Dr. Flora explains how artificial intelligence can finally match cancer's relentless ability to evolve and adapt. -
Rare Cancer Day Shines a Light on Diseases That Together Make Up 25% of All CancersRare Cancer Day takes place annually on September 23. -
With Fazirsiran Trial Underway, Takeda Seeks Answers in AATD-Related Liver DiseaseExperts believe treatments for AATD-related liver disease will be available in 5 to 10 years. -
Online and in Person, Podcaster Kimberly Ku Promotes ‘Empathetic Communications’Dr. Ku, an oncologist, hosts the podcast, “Val-You Based Care,” that emphasizes empathy and building trust in health care.
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Creating Space for the Next Question in FSGS CareA mother of 2 sons living with FSGS suggests that availability and timing plays a major role in addressing patients' questions.October 2, 2026
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A Patient With Pompe Disease Asks You to Take a Walk in His ShoesIf health care professionals could experience Pompe disease firsthand, they’d learn more than any textbook or lab result could tell them.October 2, 2026
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Are You Asking Your Patients With ATTR-CM the Right Questions?Patients with ATTR-CM regularly make adaptations to accommodate their progressive disease. Doctors could benefit from knowing about these changes.October 1, 2026
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How Patient Education Can Strengthen AATD AdvocacyWhen patients with AATD have a strong understanding of their rare disease, they can help raise awareness and better advocate for themselves.September 30, 2026
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The Complex Relationship Between SM and Hereditary Alpha-TryptasemiaHereditary alpha-tryptasemia and SM exhibit overlapping biochemical and clinical features that may present challenges for accurate diagnosis. -
Understanding Kidney Involvement in Primary Biliary CholangitisEmerging research supports a potential association between primary biliary cholangitis (PBC) and kidney disease. -
Advances in MCL Therapy Make Treatment Sequencing More CriticalMCL therapy is shifting toward targeted and immune-based approaches, making the order of treatment increasingly consequential. -
Immunosenescence in IPF: Biomarkers and Targeted TherapiesImmunosenescence offers a novel framework for IPF pathogenesis and treatment, though associated biomarkers and therapies await clinical validation. -
How Viral Infections Influence Myasthenia GravisGrowing evidence suggests viral infections can exacerbate myasthenia gravis (MG) or even contribute to its development.

- Achondroplasia
- Acromegaly
- 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
- Familial Chylomicronemia Syndrome
- Fetal and Neonatal Alloimmune Thrombocytopenia
- Focal Segmental Glomerulosclerosis
- 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
- Mantle Cell Lymphoma
- Medullary Thyroid Carcinoma
- Multiple Sclerosis
- Myasthenia Gravis
- Myelodysplastic Syndromes
- Myelofibrosis
- Neurofibromatosis Type 1
- Neuromyelitis Optica Spectrum Disorder
- Paroxysmal Nocturnal Hemoglobinuria
- Pompe Disease
- Prader-Willi Syndrome
- Primary Biliary Cholangitis
- Primary Central Nervous System Lymphoma
- Progressive Pulmonary Fibrosis
- Pulmonary Arterial Hypertension
- Sickle Cell Disease
- Spinal Muscular Atrophy
- Systemic Mastocytosis
- Systemic Sclerosis
- Thymidine Kinase 2 Deficiency
- Transthyretin Amyloid Polyneuropathy
- Transthyretin-Mediated Amyloid Cardiomyopathy
- Warm Autoimmune Hemolytic Anemia
- Wilson Disease



