Patients and HCPs Look to PFS Benefit When Choosing Treatment in CLL
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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. -
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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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 -
When Friends Doubt You Have a Rare Disease Like AATDIt can be difficult for patients with alpha-1 antitrypsin deficiency (AATD) to be doubted while dealing with the burden of disease.March 28, 2025
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Daily Challenges of Living With Pompe DiseaseEveryone with Pompe disease is diagnosed at different times in life, so the level of muscle weakness differs from person to person.March 28, 2025 -
Lessons Learned Managing Infusion Treatments for MGGetting regular infusion treatments is a common experience for patients with myasthenia gravis (MG).March 25, 2025
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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. -
The Long-Term Effects of Tafamidis on ATTR-CM Disease Progression IdentifiedA case report by Japanese authors demonstrates the strengths of tafamidis as a long-term drug in managing ATTR-CM.
- 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
