Rare Disease News, Perspectives & Resources - Rare Disease Advisor
Rare Disease News is the premier online resource for the latest rare disease news, perspectives and information.
Myelodysplastic Syndromes Life Expectancy - Rare Disease Advisor
Jul 18, 2023 · Myelodysplastic syndromes (MDS) are a diverse group of clonal abnormalities of bone marrow hematopoietic stem cells characterized by the abnormal differentiation and maturation of blood cells. The usual clinical manifestations of MDS are anemia (decreased red blood cell levels), thrombocytopenia (decreased platelet levels), and leukopenia (decreased white blood cells levels). 1
Spinal Muscular Atrophy - Rare Disease Advisor
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder that leads to progressive weakness and loss of movement. SMA results from the degeneration of alpha motor neurons in the anterior horn of the spinal cord. 1,2 Common symptoms include symmetrical muscle weakness, hypotonia, and muscular atrophy, typically of the proximal muscles and the lower extremities. 1
Spinal Muscular Atrophy - SMA - Rare Disease Advisor
Get the latest news, resources, and perspectives on spinal muscular atrophy (SMA), written specifically for HCPs who treat the disease.
Sickle Cell Disease Life Expectancy - Rare Disease Advisor
Oct 17, 2023 · Sickle cell disease (SCD) is an inherited blood disorder marked by defective hemoglobin. It causes red blood cells (RBCs) to deform into a sickle shape, which results in premature death of the RBCs, interruption of blood flow, damage to multiple tissues and organs, and an increased risk for early death.
Cold Agglutinin Disease Life Expectancy - Rare Disease Advisor
Sep 22, 2021 · Cold agglutinin disease (CAD) is a rare form of autoimmune hemolytic anemia (AIHA) in which exposure to cold provokes the body’s immune system to attack its own red blood cells, causing them to clump together and eventually leading to their premature destruction (hemolysis). 1. Typical Prognosis. CAD typically occurs in older individuals, with a slight predominance among females.
The 10 Biggest Stories in Rare Disease for 2023
Dec 13, 2023 · Dizziness is common among patients with MG, found a new Australian study published in the Journal of Clinical Neuroscience, “and warrants further investigation.”The study also found that awareness about the role of relevant allied health services and their utilization varies among patients.
Hemophilia B - Rare Disease Advisor
Background. Hemophilia B, also called factor IX deficiency or Christmas disease, is the second most common type of hemophilia.The disease was first described in the literature in 1952 in a patient named Stephen Christmas.
Self-Care Advice for People Living With a Rare Disease
Jan 30, 2024 · When you live with a rare disease, every aspect of your life is affected. I live with myasthenia gravis (MG), a rare neuromuscular disease that impacts between 40,000 and 60,000 people in the United States.. I would give 2 pieces of advice to those living with a rare disease: educate yourself as much as possible about your disease, …
About Us - Rare Disease Advisor
Read about Rare Disease Advisor here. RareDiseaseAdvisor.com is the web’s premier source of news, perspectives, and resources for clinicians who treat patients with rare diseases.The website addresses the unmet informational needs of healthcare professionals seeking the latest insights into the rare diseases they treat through fresh, original, and authoritative content.