Avapritinib Shows Long-Term Benefits for Indolent Systemic Mastocytosis
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Orthopedic Care for Achondroplasia Rapidly Evolving as New Strategies EmergeOrthopedic management plays a central role in the care of patients with achondroplasia. -
Emerging Solutions Could Make TK2d Easier to Recognize and TreatTK2d has long existed in a therapeutic void, with patients having few, if any, disease-modifying treatment options. -
SMA Continues to Challenge Patients Who Couldn’t Be Treated Early in InfancyLyza Weisman spoke at the recent MDA Clinical & Scientific Conference of no longer being able to do the 'little things' that make a life. -
PoNS Neurostimulation Device Helps to Treat Gait Problems, Patients With MS SayKerrie Walters first heard of PoNS in a chance encounter with TV personality Montel Williams, who also has MS and difficulties with gait. -
As Awareness of Huntington Grows Through Help 4 HD and Others, Hope Is BuildingLauren Holder, 39, was diagnosed with Huntington disease a year after her father died from it.
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Prioritizing Your Mental Health While Living With PBCWhen I was diagnosed with primary biliary cholangitis (PBC), I was overwhelmed.July 10, 2025 -
Sharing the CAD Journey With a Swiss-Based Neuroscience ResearcherRDA columnist with cold agglutinin disease (CAD) interviews a Swiss-based neuroscience researcher diagnosed with CAD.July 8, 2025
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Educating Medical Professionals About FNAITFor those like me, with pregnancies affected by FNAIT, detailing my health history helps physicians to understand my full health picture.July 8, 2025 -
Adopt a Complete Dietary Shift to Better Manage Pompe DiseaseUpon my diagnosis of having Pompe disease, the doctors told me very little about the disease and how it would affect me.July 8, 2025
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Emerging Priorities in Diagnosing and Managing Gaucher DiseaseCurrent evidence suggests there are 8 key areas of emerging importance in diagnosing and managing Gaucher disease. -
Biomarkers Play Key Role in Diagnosing NMOSD and Tracking Disability ProgressionBiomarkers are increasingly recognized for their use in diagnosing and tracking disability progression in NMOSD. -
Assessing PBC Prevalence and the State of Care TodayThe prevalence of PBC is expected to rise in the United States and the wider world, requiring concrete, proactive action. -
Divergence of Care Strategies Between Younger and Older Patients With HCCManagement strategies differ significantly between younger and older patients with hepatocellular carcinoma (HCC). -
FNAIT Complications and Care TodayFNAIT complications can be life-threatening; as such, important strategies must be in place to prevent the worse.
- 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
- Fabry Disease
- Fetal and Neonatal Alloimmune Thrombocytopenia
- 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
- 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
