Web8 nov. 2024 · The enzyme replacement therapy Xenpozyme for acid sphingomyelinase deficiency, originating in Sanofi’s Genzyme arm, was approved, as was Boehringer’s anti-interleukin-36 receptor monoclonal... WebIonis Pharmaceuticals presently has three that are approved for use by the FDA. These include two that treat non-neurodegenerative diseases, mipomersen targeting APOB for treating homozygous familial hypercholesterolemia and alicaforsen targeting ICAM-1 for pouchitis, and nusinersen for spinal muscular atrophy which represents one of the first …
Positive clinical data at AAN 2024 reinforces Ionis
WebTofersen (formerly IONIS-SOD1Rx), also known as BIIB067, is an investigational antisense medicine designed to inhibit the production of superoxide dismutase 1 (SOD1), which is a well understood genetic cause of amyotrophic lateral sclerosis (ALS). About SOD1-ALS. SOD1-ALS is a rare, fatal, neurodegenerative disorder caused by a mutation in the ... Web28 feb. 2024 · We earned a $35 million milestone payment from Roche, as they enrolled the first patient in the Phase 3 study of IONIS-HTTRx in patients with Huntington's disease. Biogen licensed IONIS-SOD1... raystown bus tours
Ionis (IONS) Out-licenses Hepatitis B Virus Program to Glaxo
Web31 dec. 2009 · Study Record Detail Save this study Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations (SOD-1) The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. WebIonis Pharmaceuticals and Biogen developed IONIS-SOD1Rx (BIIB067), which is designed to silence the mutated version of the SOD1 gene. The therapy is currently in clinical trials. Delivery of nerve-supporting genes Because loss of function is rare in FALS, gene therapy to replace a nonfunctional gene copy is not being widely investigated. Web17 aug. 2024 · Researchers at Biogen Inc. (NASDAQ:BIIB), Ionis Pharmaceuticals Inc. (NASDAQ:IONS) and colleagues reported data from a rat model of superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) showing that a single bolus dose of intrathecal IONIS-SOD1Rx (BIIB067) increased survival by up to 64 days compared with … simply genius pop up food covers