Alexander disease

Ionis shares significant business and pipeline progress at Innovation Day, highlighting strength of capabilities from research and technology through commercialization

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Mercoledì, Ottobre 4, 2023
Seizure, Cardiac muscle, Progress, DSM-IV codes, American Medical Association, Journal, Patient, Division, ASO, Neurology, Disease, Columbia University, Tissue, Hypertriglyceridemia, Dementia, Small RNA, Ionis Pharmaceuticals, PDUFA, Pelizaeus–Merzbacher disease, FDA, FCS, Safety, Therapeutic index, Development, Alexander disease, IONS, MECP2, Medicine, HAE, MPH, JAMA, PLP1, Hereditary angioedema, Traffic barrier, RNA, AstraZeneca, Leukodystrophy, Delivery, Phase 3, Physician, World Creativity and Innovation Day, Vaccine, Pharmaceutical industry, Medical device, Residential care, Ionis, EU, ATTR

Presentations from Ionis leaders and external physicians will highlight Ionis' comprehensive integrated research, development, and commercialization capabilities, along with key pipeline and technology highlights.

Key Points: 
  • Presentations from Ionis leaders and external physicians will highlight Ionis' comprehensive integrated research, development, and commercialization capabilities, along with key pipeline and technology highlights.
  • "Our powerful and prolific research and development engine has discovered groundbreaking medicines for people with devastating diseases.
  • We have matched this engine with a talented commercial organization poised to deliver a steady flow of wholly owned medicines to patients.
  • The event will take place at the Westin New York Grand Central from 8:00 a.m. to 3:00 p.m. Eastern Time and will also be webcast.

New data presented at AD/PD™2023 show IONIS-MAPT Rx (BIIB080) substantially reduced tau protein in patients with early-stage Alzheimer's disease

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Mercoledì, Marzo 29, 2023
Cognition, International Conference on Population and Development, Alzheimer's disease, Biogen, MAPT, Pathology, PET, Alexander disease, IONS, Brain, Angelman syndrome, Biomarker, Cerebrospinal fluid, CNS, DSM-IV codes, Messenger, Trial of the century, Back pain, Protein, ATTR, Parkinson's disease, Patient, Ionis Pharmaceuticals, AD, Headache, Death, ALS, Nerve, Conference, LTE, Safety, Medical imaging, Pharmaceutical industry, Ionis, CSF

IONIS-MAPTRx also reduced aggregated tau pathology, as measured by positron emission tomography (PET) in all brain composites assessed.

Key Points: 
  • IONIS-MAPTRx also reduced aggregated tau pathology, as measured by positron emission tomography (PET) in all brain composites assessed.
  • In patients with AD, tau protein can form "tangles" that progressively accumulate in brain regions involved in cognition.
  • It is designed to target microtubule-associated protein tau (MAPT) mRNA and prevent production of tau protein.
  • In addition to Alzheimer's disease, our clinical stage neurology programs include ATTR polyneuropathy, ALS, Alexander disease, Parkinson's disease and Angelman syndrome."

New report shows more than 260 medicines in development to fight neurodegenerative diseases

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Martedì, Ottobre 12, 2021
Pharmaceutical Research and Manufacturers of America, Brain, Risk, Amyotrophic lateral sclerosis, Biology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Rett syndrome, Multiple system atrophy, Digital, SMA, Batten disease, Spinal cord, Digital technology, MS, Central nervous system, R, Late life depression, Multimedia, Drug development, Name, Diagnosis, Neuron, Neurodegeneration, Alexander disease, Population, ALS, Alzheimer's disease, FDA, Peripheral nervous system, Ageing, Disorder, Multiple sclerosis, Palsy, Incidence, Hope, Huntington's disease, Therapy, Dementia, Spinal muscular atrophy, Friedreich's ataxia, Patient, Disease, Science, Research, Technology, Parkinson's disease, Pharmaceutical industry, Veterinary medicine, Medical device

Neurodegenerative diseases alone already cost the U.S. economy more than $655 billion a year in medical expenses and economic losses.

Key Points: 
  • Neurodegenerative diseases alone already cost the U.S. economy more than $655 billion a year in medical expenses and economic losses.
  • Today, PhRMA released a new report detailing the more than 260 medicines in development for 29 neurodegenerative diseases, all of which are in clinical trials or awaiting review by the U.S Food and Drug Administration (FDA).
  • The biopharmaceutical industry continues to work to explore new targets for earlier detection, treatments for development and ultimately, cures for neurodegenerative diseases.
  • To read the full neurodegenerative disease medicines in development report, click here .