Survey Highlights Gaps in Management of GPP in Brazil
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Dr. Katherine Mathews Honored for 4-Decade Career Researching Muscular DystrophyKatherine Mathews, MD, leads the University of Iowa's Wellstone Muscular Dystrophy Specialized Research Center. -
Nursing Home Referral Leads to a Lifetime of Huntington Disease ActivismJimmy Pollard is a former special education teacher who at one point found himself caring for 61 people with Huntington disease. -
IgG4-RD: A Mimicker of Multiple MalignanciesIn the past few years alone, researchers have reported myriad cases of IgG4-RD being mistaken for some type of malignancy. -
Boston Dermatologist Says the Most Severe Form of Psoriasis Can Be a ‘True Emergency’Generalized pustular psoriasis is the most serious form of psoriasis, with more than half of all patients with the condition needing hospitalization. -
Lifelong Care of the Patient With AchondroplasiaA polysomnography is recommended in the first year of life for patients with achondroplasia to identify abnormalities in breathing patterns.
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On the Road to Gene Therapy for HemophiliaI was offered the chance to be the first patient with hemophilia at Children's Hospital of Philadelphia to receive a new gene therapy.April 21, 2025 -
Tell Me Something About MS I Don’t KnowMost patients with multiple sclerosis don't need proof they deal with fatigue, but they would like to reduce risk and better manage fatigue.April 18, 2025
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I Am Going to WODC to Advocate on Behalf of Rare Disease PatientsI live with indolent systemic mastocytosis (SM) and it is my goal to provide hope and help to anyone living with a rare disease.April 17, 2025 -
The Consequences of Temperature Swings When Coping With CADTemperature variations can cause hemoglobin fluctuations in people with cold agglutinin disease (CAD), leading to hemolysis and other symptoms of disease.April 16, 2025
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Delving Deeper Into Quality of Life Issues Among Patients With CLLBecause of the significant disease burden of CLL, many patients struggle with issues related to health-related quality of life. -
The Potential Long-Term Effects of FNAIT on WeightResearchers continue to explore the potential long-term effects of fetal and neonatal alloimmune thrombocytopenia, such as any impact on baby weight. -
Assessing Both Patient and Physician Perspectives in Improving ATTR-PN CareTo provide holistic care for individuals with ATTR-PN, both patient and physician perspectives must be taken into account. -
Exploring the Orthopedic Burden of Achondroplasia and Its Impact on QoLPatients with achondroplasia often struggle with orthopedic complications that affect quality of life and require surgical intervention. -
The Current Treatment Landscape of HDFNWith nipocalimab currently being the sole experimental HDFN therapy, treatment relies heavily on antenatal and postnatal standard of care.
- 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
- Hepatocellular Carcinoma
- 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
