Equivocal Scans and ATTR-CM Phenotype May Signify Early ATTR-CM Diagnosis
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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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Old Friends Share Lives With Invisible, Rare DiseasesI was an avid tennis player before I was diagnosed with myasthenia gravis (MG) at 58 years old.July 2, 2025 -
Smartphones and AI: Tools to Empower the Fight Against MSPhone apps integrated with AI may be able to track, analyze, and predict multiple sclerosis (MS) progression, enabling patient empowerment.June 30, 2025
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Relapse and Setbacks: How to Be Flexible With My ITPLearning to live as an individual with immune thrombocytopenia (ITP) means learning to live with relapses and setbacks.June 30, 2025 -
Meeting the Emotional and Physical Demands of a High-Risk PregnancyKell antibodies are notoriously unpredictable compared to other alloantibody types, such as anti-C, anti-Fyª, that are associated with HDFN.June 27, 2025
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FNAIT Complications and Care TodayFNAIT complications can be life-threatening; as such, important strategies must be in place to prevent the worse. -
The Need for Tailored Tools to Assess QoL in AchondroplasiaA comprehensive review of QoL tools used in achondroplasia reveals the urgent need for more precise, validated measures. -
Elderly Patients With ATTR-CM May Benefit From Tafamidis UseTafamidis appears to be effective in extending survival even among very elderly patients with transthyretin amyloid cardiomyopathy. -
Appraising Currently Available Therapeutic Strategies in ATTR-PNResearchers appraised the strengths of currently available therapeutic strategies in managing transthyretin amyloid polyneuropathy. -
Advancing SM Diagnosis With a Standardized ProtocolThere are gaps in SM diagnostics and researchers believe there would be advantages to implementing a system-wide screening algorithm.
- 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
