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  1. Article: Anti-CGRP Monoclonal Antibodies: the Next Era of Migraine Prevention?

    Tso, Amy R / Goadsby, Peter J

    Current treatment options in neurology

    2017  Volume 19, Issue 8, Page(s) 27

    Abstract: Opinion statement: Migraine is a very disabling disorder with severe impact on patients' lives and substantive costs to society in terms of healthcare costs and lost productivity. Prevention is a key component of migraine therapy, and while numerous ... ...

    Abstract Opinion statement: Migraine is a very disabling disorder with severe impact on patients' lives and substantive costs to society in terms of healthcare costs and lost productivity. Prevention is a key component of migraine therapy, and while numerous preventive options exist, each is burdened by either troublesome side effects or insufficient efficacy. All migraine preventives currently in clinical use were licensed for other purposes and, by chance, have efficacy against migraine. As our understanding of migraine has evolved, calcitonin gene-related peptide (CGRP) has moved to the forefront as a neuropeptide central to migraine pathophysiology. Six small molecule CGRP receptor antagonists were shown to be effective for acute treatment of migraine; two were stopped for hepatotoxicity or one for formulation concern issues and one is now in phase III. Monoclonal antibodies against CGRP or the CGRP receptor have a longer duration of action and have been investigated for migraine prevention. Four are in development and three have completed phase II and one phase III trials; every reported study has been positive. Furthermore, no safety issues have arisen to date, including hepatic or cardiovascular effects, and initial tolerability appears to be excellent. Monoclonal antibodies antagonizing the CGRP pathway represent a novel approach to prevention: a mechanism-specific migraine-targeted therapy. While we must await the results of all the phase III trials, cautious excitement seems warranted as we enter a new era of better tolerated, well-understood, bespoke migraine treatment for this common and disabling neurological disorder.
    Language English
    Publishing date 2017-06-18
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 2057342-X
    ISSN 1534-3138 ; 1092-8480
    ISSN (online) 1534-3138
    ISSN 1092-8480
    DOI 10.1007/s11940-017-0463-4
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Headache.

    Tso, Amy R / Goadsby, Peter J

    Seminars in neurology

    2016  Volume 36, Issue 5, Page(s) 442–448

    Abstract: Headache disorders are primarily managed in an outpatient setting; therefore, the authors focus on the primary headache disorders that comprise the bulk of clinical practice. Red flags for secondary headaches that may be more commonly encountered in ... ...

    Abstract Headache disorders are primarily managed in an outpatient setting; therefore, the authors focus on the primary headache disorders that comprise the bulk of clinical practice. Red flags for secondary headaches that may be more commonly encountered in clinic are briefly discussed.
    MeSH term(s) Headache ; Headache Disorders/diagnosis ; Headache Disorders/therapy ; Humans
    Language English
    Publishing date 2016-09-23
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 603165-1
    ISSN 1098-9021 ; 0271-8235
    ISSN (online) 1098-9021
    ISSN 0271-8235
    DOI 10.1055/s-0036-1585450
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: Generation of Human iPSC-Derived Retinal Organoids for Assessment of AAV-Mediated Gene Delivery.

    Tso, Amy / da Costa, Bruna Lopes / Fehnel, Alexandra / Levi, Sarah R / Jenny, Laura A / Ragi, Sara D / Li, Yao / Quinn, Peter M J

    Methods in molecular biology (Clifton, N.J.)

    2022  Volume 2560, Page(s) 287–302

    Abstract: Human retinal organoids derived from induced pluripotent stem cells (iPSCs) serve as a promising preclinical model for testing the safety and efficacy of viral gene therapy. Retinal organoids recapitulate the stratified multilayered epithelium structure ... ...

    Abstract Human retinal organoids derived from induced pluripotent stem cells (iPSCs) serve as a promising preclinical model for testing the safety and efficacy of viral gene therapy. Retinal organoids recapitulate the stratified multilayered epithelium structure of the developing and maturating human retina. As such, retinal organoids are unique tools to model retinal disease and to test therapeutic interventions toward their amelioration. Here, we describe a method for the generation of human iPSC-derived retinal organoids and how they can be utilized for the assessment of recombinant adeno-associated viral (rAAV)-mediated gene delivery.
    MeSH term(s) Humans ; Induced Pluripotent Stem Cells ; Genetic Therapy
    Language English
    Publishing date 2022-11-08
    Publishing country United States
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
    ISSN 1940-6029
    ISSN (online) 1940-6029
    DOI 10.1007/978-1-0716-2651-1_27
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: Culture of Human Retinal Explants for Ex Vivo Assessment of AAV Gene Delivery.

    Wu, Wen-Hsuan / Tso, Amy / Breazzano, Mark P / Jenny, Laura A / Levi, Sarah R / Tsang, Stephen H / Quinn, Peter M J

    Methods in molecular biology (Clifton, N.J.)

    2022  Volume 2560, Page(s) 303–311

    Abstract: Due to the clinically established safety and efficacy profile of recombinant adeno-associated viral (rAAV) vectors, they are considered the "go to" vector for retinal gene therapy. Design of a rAAV-mediated gene therapy focuses on cell tropism, high ... ...

    Abstract Due to the clinically established safety and efficacy profile of recombinant adeno-associated viral (rAAV) vectors, they are considered the "go to" vector for retinal gene therapy. Design of a rAAV-mediated gene therapy focuses on cell tropism, high transduction efficiency, and high transgene expression levels to achieve the lowest therapeutic treatment dosage and avoid toxicity. Human retinal explants are a clinically relevant model system for exploring these aspects of rAAV-mediated gene delivery. In this chapter, we describe an ex vivo human retinal explant culture protocol to evaluate transgene expression in order to determine the selectivity and efficacy of rAAV vectors for human retinal gene therapy.
    MeSH term(s) Humans ; Genetic Therapy
    Language English
    Publishing date 2022-12-09
    Publishing country United States
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
    ISSN 1940-6029
    ISSN (online) 1940-6029
    DOI 10.1007/978-1-0716-2651-1_28
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: Increased rate of venous thrombosis may be associated with inpatient dihydroergotamine treatment.

    Tso, Amy R / Patniyot, Irene R / Gelfand, Amy A / Goadsby, Peter J

    Neurology

    2017  Volume 89, Issue 3, Page(s) 279–283

    Abstract: Objective: To review whether the incidence of catheter-associated venous thromboses was higher in patients receiving IV dihydroergotamine compared to lidocaine.: Methods: We retrospectively reviewed all admissions at the University of California, San ...

    Abstract Objective: To review whether the incidence of catheter-associated venous thromboses was higher in patients receiving IV dihydroergotamine compared to lidocaine.
    Methods: We retrospectively reviewed all admissions at the University of California, San Francisco Headache Center from February 25, 2008, through October 31, 2014, for age, sex, diagnosis, aura, treatment dose, type of IV line used, days with line, superficial (SVT) or deep venous thrombosis (DVT), and pulmonary embolism (PE).
    Results: A peripherally inserted central catheter (PICC) or midline catheter was placed in 315 of 589 (53%) admissions. Mean age was 38 years with a range of 6 to 79 years; 121 patients (21%) were ≤18 years old. Seventy-four percent (433 of 589) of patients were female. Of 263 dihydroergotamine admissions using a PICC or midline catheter, 19 (7.2%) had either an SVT or DVT or a PE; 2 patients were diagnosed with both DVT and PE. Of 52 lidocaine admissions using a PICC or midline catheter, none had a thrombotic event (
    Conclusions: IV dihydroergotamine treatment may be associated with an increased risk of catheter-associated venous thrombosis. A low threshold for diagnostic ultrasound investigation is appropriate because anticoagulation therapy was frequently required.
    MeSH term(s) Administration, Intravenous/adverse effects ; Administration, Intravenous/instrumentation ; Adolescent ; Adult ; Aged ; Analgesics, Non-Narcotic/administration & dosage ; Analgesics, Non-Narcotic/adverse effects ; Catheters, Indwelling/adverse effects ; Child ; Dihydroergotamine/administration & dosage ; Dihydroergotamine/adverse effects ; Female ; Humans ; Incidence ; Inpatients/statistics & numerical data ; Lidocaine/administration & dosage ; Lidocaine/adverse effects ; Male ; Middle Aged ; Multivariate Analysis ; Retrospective Studies ; Tertiary Care Centers ; Venous Thrombosis/epidemiology ; Young Adult
    Chemical Substances Analgesics, Non-Narcotic ; Dihydroergotamine (436O5HM03C) ; Lidocaine (98PI200987)
    Language English
    Publishing date 2017-07-18
    Publishing country United States
    Document type Comparative Study ; Journal Article
    ZDB-ID 207147-2
    ISSN 1526-632X ; 0028-3878
    ISSN (online) 1526-632X
    ISSN 0028-3878
    DOI 10.1212/WNL.0000000000004108
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article ; Online: Recent neuroimaging advances in the study of primary headaches.

    Tso, Amy R / Goadsby, Peter J

    Current pain and headache reports

    2015  Volume 19, Issue 6, Page(s) 15

    Abstract: Neuroimaging techniques can be used to investigate both functional and structural features of the brain in patients who have primary headache disorders such as migraine or cluster headache. Improved treatments are needed for both, and this goal will ... ...

    Abstract Neuroimaging techniques can be used to investigate both functional and structural features of the brain in patients who have primary headache disorders such as migraine or cluster headache. Improved treatments are needed for both, and this goal will likely be facilitated by a better understanding of the underlying biology. Functional imaging studies have identified regions active during attacks, as well as abnormalities that are present during the interictal period. Volumetric, surface-based morphometric, and tractography studies have revealed structural changes, although whether these represent a cause or effect of the condition remains to be determined. The development of new techniques and modalities promises to yield additional insights in the future. This article aims to review the major findings and most recent advances in neuroimaging of migraine and cluster headache.
    MeSH term(s) Brain/blood supply ; Brain/pathology ; Brain/physiopathology ; Functional Neuroimaging/trends ; Gray Matter/pathology ; Headache Disorders, Primary/diagnosis ; Headache Disorders, Primary/etiology ; Headache Disorders, Primary/physiopathology ; Humans ; Magnetic Resonance Imaging ; White Matter/pathology
    Language English
    Publishing date 2015-06
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 2055062-5
    ISSN 1534-3081 ; 1531-3433
    ISSN (online) 1534-3081
    ISSN 1531-3433
    DOI 10.1007/s11916-015-0487-y
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  7. Article ; Online: Noninvasive Vagus Nerve Stimulation for Treatment of Indomethacin-Sensitive Headaches.

    Tso, Amy R / Marin, Juana / Goadsby, Peter J

    JAMA neurology

    2017  Volume 74, Issue 10, Page(s) 1266–1267

    MeSH term(s) Adult ; Anti-Inflammatory Agents, Non-Steroidal/adverse effects ; Anti-Inflammatory Agents, Non-Steroidal/therapeutic use ; Female ; Headache/chemically induced ; Headache/diagnosis ; Humans ; Indomethacin/adverse effects ; Indomethacin/therapeutic use ; Male ; Middle Aged ; Paroxysmal Hemicrania/diagnosis ; Paroxysmal Hemicrania/therapy ; Vagus Nerve Stimulation/methods
    Chemical Substances Anti-Inflammatory Agents, Non-Steroidal ; Indomethacin (XXE1CET956)
    Language English
    Publishing date 2017-08-28
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2702023-X
    ISSN 2168-6157 ; 2168-6149
    ISSN (online) 2168-6157
    ISSN 2168-6149
    DOI 10.1001/jamaneurol.2017.2122
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article: New targets for migraine therapy.

    Tso, Amy R / Goadsby, Peter J

    Current treatment options in neurology

    2014  Volume 16, Issue 11, Page(s) 318

    Abstract: Opinion statement: The shift in our understanding of migraine as a vascular disorder to a brain disorder has opened new avenues for the development of novel therapeutics with neural targets. The advent of 5-HT1B/1D receptor agonists, the triptans, in ... ...

    Abstract Opinion statement: The shift in our understanding of migraine as a vascular disorder to a brain disorder has opened new avenues for the development of novel therapeutics with neural targets. The advent of 5-HT1B/1D receptor agonists, the triptans, in the 1990s was a crucial step in the modern evolution of treatment. The use of triptans, like their predecessors, is limited by their vasoconstrictor effects, and new development has been slowed by poor academic research funding to identify new targets. The development of agents without vascular effects, such as calcitonin gene-related peptide receptor antagonists and selective serotonin 5-HT1F receptor agonists, will bring more effective treatments to a population currently without migraine-specific options. In addition, advances in understanding migraine pathophysiology have identified new potential pharmacologic targets such as acid-sensing ion channels, glutamate and orexin receptors, nitric oxide synthase (NOS), and transient receptor potential (TRP) channels. Although previous attempts to block subtypes of glutamate receptors, NOS, and TRP channels have had mixed outcomes, new molecules for the same targets are currently under investigation. Finally, an entirely new approach to migraine treatment with noninvasive neuromodulation via transcutaneous neurostimulation or transcranial magnetic stimulation is just beginning. Hopefully in the coming years we will see a new era of migraine therapy, with multiple classes of better-tolerated, more effective agents targeting diverse yet specific migraine mechanisms.
    Language English
    Publishing date 2014-09-20
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2057342-X
    ISSN 1534-3138 ; 1092-8480
    ISSN (online) 1534-3138
    ISSN 1092-8480
    DOI 10.1007/s11940-014-0318-1
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article: Headache

    Tso, Amy R. / Goadsby, Peter J.

    Seminars in Neurology

    (Pearls and Pitfalls, Part 2)

    2016  Volume 36, Issue 05, Page(s) 442–448

    Abstract: Headache disorders are primarily managed in an outpatient setting; therefore, the authors focus on the primary headache disorders that comprise the bulk of clinical practice. Red flags for secondary headaches that may be more commonly encountered in ... ...

    Series title Pearls and Pitfalls, Part 2
    Abstract Headache disorders are primarily managed in an outpatient setting; therefore, the authors focus on the primary headache disorders that comprise the bulk of clinical practice. Red flags for secondary headaches that may be more commonly encountered in clinic are briefly discussed.
    Keywords headache ; diagnosis ; treatment ; migraine
    Language English
    Publishing date 2016-09-23
    Publisher Thieme Medical Publishers
    Publishing place Stuttgart ; New York
    Document type Article
    ZDB-ID 603165-1
    ISSN 1098-9021 ; 0271-8235
    ISSN (online) 1098-9021
    ISSN 0271-8235
    DOI 10.1055/s-0036-1585450
    Database Thieme publisher's database

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  10. Article ; Online: Aprepitant for the management of nausea with inpatient IV dihydroergotamine.

    Chou, Denise E / Tso, Amy R / Goadsby, Peter J

    Neurology

    2016  Volume 87, Issue 15, Page(s) 1613–1616

    Abstract: Objective: To assess the efficacy and tolerability of oral aprepitant, a substance P/neurokinin A receptor antagonist, in controlling nausea associated with IV dihydroergotamine (DHE) administered for medically refractory migrainous headache in patients ...

    Abstract Objective: To assess the efficacy and tolerability of oral aprepitant, a substance P/neurokinin A receptor antagonist, in controlling nausea associated with IV dihydroergotamine (DHE) administered for medically refractory migrainous headache in patients not responding to standard antiemetics or with a history of uncontrolled nausea with DHE.
    Methods: This was a retrospective chart review of prospectively collected hourly diary data and clinical notes of patients hospitalized between 2011 and 2015 for inpatient treatment with DHE. Patients were classified using the International Classification of Headache Disorders, 3rd edition (beta version). Peak and average daily nausea scores from hourly diaries, or daily entries of notes, and concurrent antiemetic use were collected and tabulated.
    Results: Seventy-four patients, of whom 24 had daily diaries, with chronic migraine with or without aura, with or without medication overuse, or new daily persistent headache of a migrainous type, were identified. In 36 of 57 cases in which aprepitant was administered during hospitalization, there was a 50% reduction in the average daily number of as-needed antinausea medications. Of 57 patients, 52 reported that the addition of aprepitant improved nausea. Among 21 of 24 patients with hourly diary data, nausea scores were reduced and in all 12 with vomiting there was cessation of emesis after aprepitant was added. Aprepitant was well tolerated with no treatment emergent adverse events.
    Conclusions: Aprepitant can be effective in the treatment of refractory DHE-induced nausea and emesis. Given the broader issue of troublesome nausea and vomiting in acute presentations of migraine, general neurologists may consider what place aprepitant has in the management of such patients.
    Classification of evidence: This study provides Class IV evidence that for patients with medically refractory migraine receiving IV DHE, oral aprepitant reduces nausea.
    MeSH term(s) Administration, Intravenous ; Administration, Oral ; Analgesics, Non-Narcotic/administration & dosage ; Analgesics, Non-Narcotic/adverse effects ; Antiemetics/therapeutic use ; Dihydroergotamine/administration & dosage ; Dihydroergotamine/adverse effects ; Humans ; Inpatients ; Migraine Disorders/drug therapy ; Morpholines/therapeutic use ; Nausea/drug therapy ; Nausea/etiology ; Prospective Studies ; Retrospective Studies ; Treatment Outcome
    Chemical Substances Analgesics, Non-Narcotic ; Antiemetics ; Morpholines ; aprepitant (1NF15YR6UY) ; Dihydroergotamine (436O5HM03C)
    Language English
    Publishing date 2016-10-11
    Publishing country United States
    Document type Journal Article
    ZDB-ID 207147-2
    ISSN 1526-632X ; 0028-3878
    ISSN (online) 1526-632X
    ISSN 0028-3878
    DOI 10.1212/WNL.0000000000003206
    Database MEDical Literature Analysis and Retrieval System OnLINE

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