ANCA-Associated Vasculitis Life Expectancy - Rare Disease Advisor
ANCA-associated vasculitis (AAV) is a rare autoimmune disease that can cause inflammation and damage in various organs and tissues. The life expectancy of patients with AAV depends on several factors, such as disease subtype, onset, treatment, and complications.
Lennox-Gastaut Syndrome Life Expectancy - Rare Disease Advisor
Lennox-Gastaut syndrome (LGS) is a severe form of childhood epilepsy with poor prognosis and high mortality. Learn about the factors affecting life expectancy, such as seizure control, brain injuries, SUDEP, and accidents, and the studies on survival rates in LGS.
Systemic Sclerosis Life Expectancy - Rare Disease Advisor
Learn how systemic sclerosis (SSc) affects life expectancy and what factors influence survival. Find out the mortality rates, prognostic factors, and complications of SSc subtypes and organ involvement.
IgG4-Related Disease Life Expectancy - Rare Disease Advisor
IgG4-related disease (IgG4-RD) is a rare, immune-mediated, fibroinflammatory condition that may affect multiple organs and increase the risk of death. Learn about the factors that affect the prognosis of IgG4-RD, such as age, renal dysfunction, malignancy, and multiorgan involvement.
Pompe Disease History - Rare Disease Advisor
Pompe disease, also known as acid maltase deficiency or glycogen storage disease type II, is a rare genetic disorder characterized by a deficiency or absence of the lysosomal acid alpha-glucosidase enzyme (GAA).GAA normally cleaves glycogen to form glucose. Abnormalities of the GAA enzyme due to mutations in the GAA gene result in an accumulation of glycogen in tissues, especially cardiac and ...
Muscular Dystrophy History - Rare Disease Advisor
Learn about the history of muscular dystrophy, from its first description by Sir Charles Bell in 1830 to the latest treatments and research. Find out who discovered the DMD gene, the types of muscular dystrophy, and the challenges of finding a cure.
Myasthenia Gravis Prognosis - Rare Disease Advisor
Myasthenia gravis (MG) is a rare autoimmune disease in which the body attacks components of the neuromuscular junction (NMJ), causing weakness of voluntary skeletal muscles and fatigue with repetitive movements. MG especially affects the eyes, peripheral extremities, bulbar system, and respiratory system. 1 General Prognosis. In general, most individuals with MG who receive the appropriate ...
First Patient Dosed in Phase 3 Study of Fazirsiran for AATD-LD
Apr 4, 2023 · Fazirsiran is a drug candidate that targets the genetic mutation causing AATD-LD, a rare and progressive condition that affects the liver and lungs. The first patient has been dosed in a phase 3 trial of fazirsiran, which could lead to a $40 million payment from Takeda to Arrowhead.
Distinguishing Between Guillain-Barré Syndrome and CIDP …
Jun 21, 2024 · Both Guillain-Barré syndrome (GBS) and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) are 2 disorders that belong under the umbrella of inflammatory neuropathies. Both these disorders are characterized by motor and sensory dysfunction that can give way to disability.
Systemic Mastocytosis Life Expectancy - Rare Disease Advisor
Apr 11, 2024 · Learn how the life expectancy of patients with systemic mastocytosis (SM) varies depending on the subtype, from indolent to aggressive. Find out the factors that affect survival, the treatments that improve outcomes, and the sources of data and studies.