FDA Grants Orphan Drug Designation to VAS-101 for Sickle Cell Disease
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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. -
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.
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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 -
Are Stem Cell Transplants the New Hope for Treating NMO?As someone living with neuromyelitis optica (NMO), my initial reaction to the words, “stem cell transplant,” is to run in the opposite direction.June 27, 2025
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A Pompe Turning Point: Needing a WheelchairI went years without being on Pompe treatment, and walking became my most noticeable struggle.June 24, 2025 -
Ways to Make Life With Pompe EasierThe most common challenge that comes with living with Pompe disease is getting tired more easily.June 24, 2025
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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. -
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.
- 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
