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Share this article Share this article CARLSBAD, Calif., April 5, 2021 /PRNewswire/ Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the initiation of a Phase 3 clinical trial of ION363 in patients with amyotrophic lateral sclerosis (ALS) with mutations in the fused in sarcoma gene ( FUS). Patients with a mutation in the FUS gene develop a rare form of ALS, referred to as FUS-ALS, which is the most common cause of juvenile-onset ALS. There is substantial evidence that mutations in the FUS gene are responsible for a toxic gain of function that can lead to rapid, progressive loss of motor neurons in patients with FUS-ALS. ION363 is an investigational antisense medicine targeting the FUS RNA to reduce the production of the FUS protein. Antisense-mediated reduction of mutant FUS protein in a FUS-ALS mouse model prevents motor neuron loss. By targeting the root cause of FUS-ALS, ION363 has the potential to reduce or prevent disease progression in FUS-ALS patients.

United statesUnited kingdomJaci hermstadC frank bennettNeil shneiderPrnewswire ionis pharmaceuticals incDrug administrationIonis pharmaceuticals incIonis pharmaceuticalsColumbia university eleanorColumbia universityLou gehrigஒன்றுபட்டது மாநிலங்களில்ஒன்றுபட்டது கிஂக்டம்கொலம்பியா பல்கலைக்கழகம் எலினோர்கொலம்பியா பல்கலைக்கழகம்