NMOSD

Horizon Therapeutics plc Ranks First in Patient Centricity and Integrity by Patient Advocacy Groups According to New PatientView Survey

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星期二, 五月 2, 2023
Biotechnology, Pharmaceutical, Health, TED, Rarey technique, Partnership, Exhibition, Education, Arthritis, Graves' ophthalmopathy, NMO, Clinical trial, Human, NMOSD, Congress, Rare, Patient, Scholarship, Horizon, Disease, Pharmaceutical industry, Nursing, Vaccine, Neurology

Horizon Therapeutics plc (Nasdaq: HZNP) today announced it ranked first in patient centricity, integrity, ease of relations and transparency in pricing by patient groups who worked with the company around the world.

Key Points: 
  • Horizon Therapeutics plc (Nasdaq: HZNP) today announced it ranked first in patient centricity, integrity, ease of relations and transparency in pricing by patient groups who worked with the company around the world.
  • The results are based on PatientView’s annual survey of more than 2,200 patient groups evaluating more than 40 biotechnology and pharmaceutical companies.
  • Among patient groups familiar, but not working directly with Horizon, the company also ranked first in patient centricity, integrity and ease of relations and third overall in overall corporate reputation.
  • “Everything we do at Horizon is guided by people living with challenging diseases and the patient groups who serve them,” said Matt Flesch, vice president, product communications and patient advocacy, Horizon.

Genentech Announces Industry-Leading Brain Health Research Collaborations and Latest Data Across Neuroscience Medicines at AAN 2023 Annual Meeting

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星期一, 四月 24, 2023
Mental Health, Health, Telemedicine, Virtual Medicine, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, DSM-IV codes, Spinal muscular atrophy, Survival, Multiple sclerosis, Science, Patient, Nervous system, Ageing, Policy, IHME, Disease, Maintenance, Cleveland Clinic, University, Disability, Cardiovascular disease, Dementia, Brain, NMOSD, Research, Satralizumab, RO, RMS, RRMS, Cleveland, Immunodeficiency, Phase, Death, Safety, Huntington's disease, Abstract, Natural history, COVID-19, Multimedia, ROG, Brainpower, Neuroscience, Myasthenia gravis, Cancer, EMPOWER, Angelman syndrome, SMA, Experience, Telehealth, Partnership, Central nervous system, AAN, American Academy, Digital technology, Annual general meeting, Neuromyelitis optica spectrum disorder, Tenecteplase, AD, Pharmaceutical industry, Medical imaging, Risdiplam, Genentech

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), is announcing three new partnerships focused on improving brain health outcomes.

Key Points: 
  • Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), is announcing three new partnerships focused on improving brain health outcomes.
  • “With that in mind, we are committed to developing and delivering new medicines that address areas of highest need in neuroscience.
  • See AAN oral presentation details on Genentech’s telehealth pilot with Cleveland Clinic here , Tuesday, April 25 at 3:54 p.m.
  • To meet the needs of people living with brain health conditions, Genentech is continuing to grow and advance its pipeline, with key areas of focus in neuroimmunologic, neuromuscular, neurodegenerative and neurodevelopmental diseases.

Horizon Therapeutics plc Announces UPLIZNA® (inebilizumab-cdon) Data in Neuromyelitis Optica Spectrum Disorder (NMOSD) to be Presented at the 2023 American Academy of Neurology (AAN) Annual Meeting

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星期四, 四月 20, 2023
Health, Neurology, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, ANVISA, AAN, Safety, Food, Efficacy, American Academy, Cell, Immunosuppression, B cell, Brazilian Health Regulatory Agency, FDA, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, European Commission, Pharmaceutical industry, Vaccine, NMOSD, Inebilizumab

Horizon Therapeutics plc (Nasdaq: HZNP) today announced that multiple UPLIZNA analyses will be presented at the 2023 American Academy of Neurology (AAN) Annual Meeting, April 22-27 in Boston.

Key Points: 
  • Horizon Therapeutics plc (Nasdaq: HZNP) today announced that multiple UPLIZNA analyses will be presented at the 2023 American Academy of Neurology (AAN) Annual Meeting, April 22-27 in Boston.
  • UPLIZNA is the first and only anti-CD19 B-cell-depleting humanized monoclonal antibody approved by the U.S. Food and Drug Administration (FDA), European Commission (EC) and Brazilian Health Regulatory Agency (ANVISA) for the treatment of NMOSD in adults who are anti-aquaporin-4-immunoglobulin G seropositive (AQP4-IgG+).
  • “Of note, the oral presentation of data on B-cell subsets and aquaporin-4 antibody titers will shine a light on the role of plasmablasts and plasma cells in NMOSD attacks, and in turn, how targeting these cells with treatment may help prevent attacks.
  • A deeper understanding of the mechanism of this devastating disease will continue to enhance our ability to improve patient outcomes.”

The SPHERES Registry of Patients with NMOSD Reaches Enrollment Milestone of 200 Patients

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星期三, 四月 12, 2023
David Geffen School of Medicine at UCLA, Pros, Central nervous system, Therapy, Registry, Disability, NMO, MFIS, Spinal cord, Aes, Quality of life, Research, Brain, Biomarker, Experiment, Synergy, Disability studies, SPHERES, Patient, Physician, Collection, Cranial nerves, Neuromyelitis optica spectrum disorder, Diagnosis, UCLA, FDA, Disease, Autoimmunity, Safety, Inflammation, Pharmaceutical industry, Medicine, NMOSD

The SPHERES Registry has recruited 200 patients, across 23 sites in North America, supported by 72 physicians.

Key Points: 
  • The SPHERES Registry has recruited 200 patients, across 23 sites in North America, supported by 72 physicians.
  • Launched in early 2021, the SPHERES Registry serves as the first regulatory-grade and real-world study to advance the understanding of NMOSD.
  • To date, the SPHERES Registry has recruited 200 patients, across 23 sites in North America, supported by 72 physicians.
  • Today, advances in diagnosis, biomarkers, and newly FDA-approved treatment options offer patients with NMOSD the promise to lessen or prevent disability and enhance quality of life.

Devic's Syndrome Treatment Global Market Report 2022: Soliris (Eculizumab) to Foster Growth in the Sector - ResearchAndMarkets.com

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星期三, 三月 22, 2023
Health, Pharmaceutical, Complement component 5, Neuron, Syndrome, C5a, Spinal cord, Neuromyelitis optica spectrum disorder, National Multiple Sclerosis Society, MedImmune, Chugai Pharmaceutical Co., Woman, NMOSD, CNS, Plasma protein binding, Protein, Patient, Immune system, Optic nerve, Alexion, Diagnosis, Rare, Death, Autoantibody, Pharmaceutical industry, Eculizumab

Soliris works by specifically preventing activation of particular proteins in the complement system (C5a and C5b), which play a role in the treatment of Devic s syndrome.

Key Points: 
  • Soliris works by specifically preventing activation of particular proteins in the complement system (C5a and C5b), which play a role in the treatment of Devic s syndrome.
  • Patients suffering with Devic's syndrome possess AQP4 antibody-positive, the body s immune system can turn against itself to produce autoantibodies against AQP4.
  • There are three promising molecules in Devic s syndrome treatment pipeline such as Satralizumab - Phase 3 (Hoffmann-La Roche/ Chugai Pharmaceutical), MEDI-551- Phase 2/3 (MedImmune LLC), and RC18 - Phase 3 (RemeGen, Ltd.).
  • According to National Multiple Sclerosis Society, there are around 4,000 people suffering with Devic's syndrome in the United States with majority of women population.

New Analysis of MRI Findings Show UPLIZNA® (inebilizumab-cdon) Reduced the Formation of Asymptomatic Optic Nerve Lesions in People With Neuromyelitis Optica Spectrum Disorder (NMOSD)

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星期二, 三月 14, 2023
Research, Neurology, FDA, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Optical, Science, MAS, ANVISA, NMO, NMOSD, Prognosis, Regulation of tobacco by the U.S. Food and Drug Administration, Risk, MRI, OLP, Patient, RCP, Optic nerve, European Commission, Spinal cord, Brazilian Health Regulatory Agency, Optic neuritis, Food, University, Civil service commission, Medical imaging

This new post-hoc analysis based on MRI imaging provides a deeper understanding of the treatment effect on subclinical lesions.

Key Points: 
  • This new post-hoc analysis based on MRI imaging provides a deeper understanding of the treatment effect on subclinical lesions.
  • The MRI findings showed that a high proportion of participants experienced subclinical optic nerve findings without new symptoms at the end of the RCP.
  • Importantly, while the total subclinical findings in the optic nerve were not reduced during the RCP, the number of clinical optic neuritis attacks was significantly reduced during this time period.
  • Moreover, the formation of these subclinical findings was reduced with repeated UPLIZNA treatment over the course of the OLP.

Ad Scientiam Launches Programs to Develop Digital Biomarkers for Chronic Neurological Diseases

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星期二, 二月 28, 2023
Spectrum disorder, Total permanent disability insurance, Collection, Disease, Self-assessment, Biomarker, Research, Algorithm, Patient, Rare disease, Digital, GMG, Fatigue, Alexion, Communication, Pharmaceutical industry, Medical imaging, NMOSD, Neurology

The programs will leverage assets, methodologies and expertise built by Ad Scientiam over the past five years and are supported by Alexion, AstraZeneca Rare Disease.

Key Points: 
  • The programs will leverage assets, methodologies and expertise built by Ad Scientiam over the past five years and are supported by Alexion, AstraZeneca Rare Disease.
  • gMG and NMOSD are chronic severe diseases in which progression is complex and critical to monitor.
  • Digital biomarkers are patient-generated physiological and behavioral measures that are collected from digital devices like smartphones and processed by algorithms.
  • Scoping phases have been conducted for both programs to identify clinically meaningful digital biomarkers to monitor patients living with NMOSD or gMG.

Ad Scientiam Launches Programs to Develop Digital Biomarkers for Chronic Neurological Diseases

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星期二, 二月 28, 2023
Spectrum disorder, Total permanent disability insurance, Collection, Disease, Self-assessment, Biomarker, Research, Algorithm, Patient, Rare disease, Digital, GMG, Fatigue, Alexion, Communication, Pharmaceutical industry, Medical imaging, NMOSD, Neurology

The programs will leverage assets, methodologies and expertise built by Ad Scientiam over the past five years and are supported by Alexion, AstraZeneca Rare Disease.

Key Points: 
  • The programs will leverage assets, methodologies and expertise built by Ad Scientiam over the past five years and are supported by Alexion, AstraZeneca Rare Disease.
  • gMG and NMOSD are chronic severe diseases in which progression is complex and critical to monitor.
  • Digital biomarkers are patient-generated physiological and behavioral measures that are collected from digital devices like smartphones and processed by algorithms.
  • Scoping phases have been conducted for both programs to identify clinically meaningful digital biomarkers to monitor patients living with NMOSD or gMG.

One Year Post-Launch of Argenx's Vyvgart for gMG, US Neurologists Confused on Best Approach to Treatment Dosing Schedules, According to Spherix Global Insights

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星期五, 二月 24, 2023
Neurology, PDUFA, Genentech, UCB, Research, Neonatal Fc receptor, NMOSD, Patient, NASA RealWorld-InWorld Engineering Design Challenge, Inebilizumab, Market, Janssen, GMG, Hatred, Launch, Nursing, Pharmaceutical industry, Efgartigimod alfa, Ravulizumab, EU

EXTON, Pa., Feb. 24, 2023 /PRNewswire/ -- Argenx's launch of Vyvgart (efgartigimod) has been a success, with global first-year revenue of ~$400 million USD. As the second biologic approved for generalized myasthenia gravis (gMG) and the first neonatal Fc receptor (FcRn) blocker, Vyvgart's novel and targeted mechanism of action has resonated with HCPs. Indeed, neurologists and neuromuscular specialists surveyed by Spherix Global Insights consider Vyvgart to address a substantial unmet in the treatment of this chronic, rare autoimmune condition.

Key Points: 
  • Indeed, neurologists and neuromuscular specialists surveyed by Spherix Global Insights consider Vyvgart to address a substantial unmet in the treatment of this chronic, rare autoimmune condition.
  • Despite these positive perceptions of argenx's brand, surveyed respondents (n=69) remain less confident in Vyvgart's dosing profile.
  • Interestingly, even with increased experience among neurologists, half continue to report uncertainty as to when to re-dose patients after the first Vyvgart treatment cycle.
  • Indeed, when probed unaided on Vyvgart's biggest disadvantages, two in five offered its dosing profile as a leading disadvantage.

New Data Suggest UPLIZNA® (inebilizumab-cdon) for the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD) Did Not Increase the Risk of COVID-19 Infection or Reduce Antibody Levels From Childhood Vaccines

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星期四, 二月 23, 2023
Health, Other Health, COVID-19, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Tetanus, Safety, Civil service commission, Learning, Doctor of Philosophy, Patient, University, Research, Neuroscience, Treatment, Neurology, Risk, Deep vein thrombosis, Great America Committee, NMOSD, Mumps, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, MAS, Multiple sclerosis, Infection, History, Rubella, Pulmonary embolism, Varicella zoster virus, Fatality, Measles, European Commission, Incidence, Neuromyelitis optica spectrum disorder, Food, Vaccine, COVID, Pharmaceutical industry

These data suggest UPLIZNA does not increase susceptibility to COVID infection or deplete childhood vaccine-generated antibodies.

Key Points: 
  • These data suggest UPLIZNA does not increase susceptibility to COVID infection or deplete childhood vaccine-generated antibodies.
  • In total, the analysis found a low incidence rate of infections (0.024 events per patient year) among UPLIZNA-treated patients.
  • Of the 10 patients with known outcomes, six were reported as “recovered/resolved,” two as not recovered/resolved and two patients died.
  • “The results of these analyses provide further support that UPLIZNA is an effective long-term treatment for NMOSD with a favorable safety profile.”