Phase 3b Trial Supports Direct Transition to Mim8 in Hemophilia A
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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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Hemophilia: Knowing Your Options and Trusting Your TeamRecently, I had the opportunity to attend different conferences to raise awareness about hemophilia and its treatment options.July 7, 2025 -
Living With AATD and Managing Patient BurnoutIndividuals with rare diseases, such as alpha-1 antitrypsin deficiency (AATD), can easily experience patient burnout and frustration.July 3, 2025
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“Finding Nemo” Offers Lessons for Living With a DisabilityI watched 'Nemo" repeatedly, and yet I didn’t realize it was a movie inherently about disability until I got sick with CIDP.July 3, 2025 -
Then and Now: What Life With Friedreich Ataxia Is LikeThose trips were pre-Friedreich ataxia (FA), when life was so much simpler in so many different ways.July 3, 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
