Ionis alexander disease
Web11 feb. 2024 · CARLSBAD, Calif., Feb. 11, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that … Web20 apr. 2024 · Alexander disease is a rare neurological condition characterized as a leukodystrophy, or a disease affecting the myelin sheath (the fatty insulation that …
Ionis alexander disease
Did you know?
WebAlong the way she earned a Ph.D. at the University of Washington and completed her postdoctoral studies at Harvard University. At Ionis, she leads research on investigational medicines to treat Alexander disease and Pelizaeus-Merzbacher disease, two rare neurodevelopmental disorders affecting young children. Web20 apr. 2024 · Alexander disease is a rare neurological condition characterized as a leukodystrophy, or a disease affecting the myelin sheath (the fatty insulation that protects a nerve fiber and supports...
Web18 jan. 2024 · These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and the most recent Form 10-Q ... WebTo determine if the pharmacokinetics and pharmacodynamics of gapmer antisense oligonucleotides (ASOs), containing phosphorothioate backbones and 2'-O-methoxyethyl RNA modifications (2'-MOE ASOs), can be altered by renal disease, a series of experiments were performed in models of chronic kidney disease (CKD) and acute kidney injury (AKI).
WebIONIS-FXI RX (BAY2306001) is an antisense oligonucleotide that inhibits the synthesis of coagulation factor XI (FXI) and has been investigated in healthy volunteers and patients with end-stage renal disease (ESRD). FXI-LICA (BAY2976217) shares the same RNA sequence as IONIS-FXI RX … WebIn each episode of Ionis Innovations podcast, our host Dave Ecker and his guests will explore the fascinating field of drug discovery. Skip ... she leads research on investigational medicines to treat Alexander disease and Pelizaeus-Merzbacher disease, two rare neurodevelopmental disorders affecting young children. Elaine Pirie, Ph.D ...
WebIONIS-MAPT Rx is being developed to treat people with Alzheimer’s disease (AD) and potentially other neurodegenerative disorders characterized by the deposition of abnormal tau protein in the …
Web17 nov. 2024 · People born with Alexander disease may develop an enlarged brain and head, experience seizures or delayed development, have stiffness in their arms and legs, … immaculate phonk 1 hourWeb5 apr. 2024 · Advancement of ION363 to a pivotal trial is the latest example of the power of Ionis' antisense technology to potentially target the root causes of neurological diseases," said C. Frank Bennett ... list of scythiansWeb31 dec. 2024 · Alexander disease affects approximately 500 people in the EU. It is caused by a mutation in a protein that creates overproduction of glial fibrillary acidic protein (GFAP) in the brain. ION373 is designed to stop the mutated gene from producing excess GFAP. list of scudetto winnersWeb2 okt. 2024 · Developed by Ionis, ION373 is an investigational antisense medicine, designed to stop the mutated gene from producing excess glial fibrillary acidic protein … list of scrypt coinsWeb31 dec. 2024 · Alexander disease affects approximately 500 people in the EU. It is caused by a mutation in a protein that creates overproduction of glial fibrillary acidic protein … list of scunthorpe united managersWeb19 aug. 2024 · A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients With Alexander Disease Actual Study Start Date : Jun 1, 2024 Anticipated Primary Completion Date : Feb 1, 2024 Anticipated Study Completion … list of scrum meetingsWeb15 jan. 2024 · Ionis Pharmaceuticals, Inc. Dec 2024 - Oct 202411 months Carlsbad, California Lead structural and biochemical efforts in the area … list of scuba gear