Vosoritide Effective in Portuguese Children With Achondroplasia
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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. -
Myasthenia Gravis, a Rare Disease, Is Rarer Still When It Strikes ChildrenTeenagers account for an estimated 10% to 15% of all MG cases in Europe and North America. -
Pioneering Case Illustrates the Promise of Personalized Gene Editing for Rare DiseasesKJ Muldoon has been achieving developmental milestones, but his long-term prognosis is unknown.
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When to Discontinue MS Disease-Modifying TherapiesDecisions about the treatment of older people with multiple sclerosis (MS) may be changing according to new research.June 20, 2025 -
Dealing With AATD Anxiety: Lessons LearnedPatients with rare diseases like alpha-1 antitrypsin deficiency (AATD) have to learn ways to manage uncertainty and accompanying anxiety.June 20, 2025
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Learning How Medical Language Matters Through My CIDP DiagnosisOf course, getting sick with chronic inflammatory demyelinating polyneuropathy (CIDP) forced me to address why I felt this way.June 16, 2025 -
MG Month Reminder: Advocacy Takes a Front SeatI want every patient who presents with myasthenia gravis (MG) symptoms to be heard.June 16, 2025
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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. -
Exploring the Factors That Impact Prognosis in NMOSDAn early age of disease onset and the early initiation of treatment are both associated with an improved prognosis in NMOSD. -
Diagnosis of CIDP Revised to Leprosy NeuropathyA patient initially received a diagnosis of CIDP, but it was later reviewed to leprosy neuropathy. -
Exploring the Use of Spesolimab in Managing Chronic GPPThe recent availability of spesolimab has transformed the treatment landscape for patients with chronic generalized pustular psoriasis (GPP). -
Stratifying Risk and Predicting Prognosis in CLLThe ability of clinicians to stratify risk and predict prognosis in chronic lymphocytic leukemia (CLL) has improved by leaps and bounds.
- 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
