Ionis Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development ofRNA-targeted therapies
By leveraging its proprietary antisense technology, Ionis focuses on treating a range of serious diseases, including neurological disorders, cardiovascular conditions, and rare diseases. The company's innovative approach allows for precise targeting of specific genes to modulate protein production, providing potential treatment options for patients with unmet medical needs. With a robust pipeline of therapies in various stages of clinical development, Ionis is dedicated to advancing the field of genetic medicine and improving patient outcomes.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it will present additional data from the pivotal Phase 3 OASIS and OASISplus studies, as well as three year data from the Phase 2 open-label extension (OLE) study of donidalorsen, the company’s investigational RNA-targeted prophylactic medicine for hereditary angioedema (HAE). Results will be presented at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress in San Diego, California.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results for the fourth quarter and full year ended December 31, 2024.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 19th at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2024 financial results.
Applications are based on data from the DEVOTE study, which demonstrate the potential for the investigational higher dose regimen of nusinersen to advance the treatment of SMA
Denali's DNL343 missed efficacy goals in the HEALEY ALS trial but was well-tolerated. Analysts await detailed biomarker data and subgroup analyses in 2025.
William Blair starts coverage on Denali Therapeutics with an Outperform rating, citing its transport vehicle platform and promising therapies for neurodegenerative diseases.
CAMBRIDGE, Mass., Oct. 08, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced detailed results from Part B and Part C of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of an investigational higher dose regimen of nusinersen in spinal muscular atrophy (SMA), showing benefits in both individuals previously treated and treatment-naïve to nusinersen with infantile-onset or later-onset SMA. The investigational, higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months, compared to the approved nusinersen regimen (SPINRAZA®). Data to be presented during the World Muscle Society (WMS) 2024 Congress (Oct. 8-12, 2024 in Prague) highlight the potential of this investigational higher dose regimen to help address remaining unmet need in SMA.
BOSTON, Sept. 04, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQPRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that that it will present preclinical and clinical data from three of its epilepsy programs at the International League Against Epilepsy (ILAE) 15th European Epilepsy Congress (EEC), being held from September 7 to 11, 2024 in Rome, Italy.
