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1.
Lancet Glob Health ; 12(4): e697-e706, 2024 Apr.
Article in English | MEDLINE | ID: mdl-38485433

ABSTRACT

Neurological conditions are the leading cause of death and disability combined. This public health crisis has become a global priority with the introduction of WHO's Intersectoral Global Action Plan on Epilepsy and Other Neurological Disorders 2022-2031 (IGAP). 18 months after this plan was adopted, global neurology stakeholders, including representatives of the OneNeurology Partnership (a consortium uniting global neurology organisations), take stock and advocate for urgent acceleration of IGAP implementation. Drawing on lessons from relevant global health contexts, this Health Policy identifies two priority IGAP targets to expedite national delivery of the entire 10-year plan: namely, to update national policies and plans, and to create awareness campaigns and advocacy programmes for neurological conditions and brain health. To ensure rapid attainment of the identified priority targets, six strategic drivers are proposed: universal community awareness, integrated neurology approaches, intersectoral governance, regionally coordinated IGAP domestication, lived experience-informed policy making, and neurological mainstreaming (advocating to embed brain health into broader policy agendas). Contextualised with globally emerging IGAP-directed efforts and key considerations for intersectoral policy design, this novel framework provides actionable recommendations for policy makers and IGAP implementation partners. Timely, synergistic pursuit of the six drivers might aid WHO member states in cultivating public awareness and policy structures required for successful intersectoral roll-out of IGAP by 2031, paving the way towards brain health for all.


Subject(s)
Global Health , Health Policy , Humans , Policy Making , Public Health , Brain
2.
Epilepsia ; 65(3): 533-541, 2024 Mar.
Article in English | MEDLINE | ID: mdl-38279786

ABSTRACT

A variety of terms, such as "antiepileptic," "anticonvulsant," and "antiseizure" have been historically applied to medications for the treatment of seizure disorders. Terminology is important because using terms that do not accurately reflect the action of specific treatments may result in a misunderstanding of their effects and inappropriate use. The present International League Against Epilepsy (ILAE) position paper used a Delphi approach to develop recommendations on English-language terminology applicable to pharmacological agents currently approved for treating seizure disorders. There was consensus that these medications should be collectively named "antiseizure medications". This term accurately reflects their primarily symptomatic effect against seizures and reduces the possibility of health care practitioners, patients, or caregivers having undue expectations or an incorrect understanding of the real action of these medications. The term "antiseizure" to describe these agents does not exclude the possibility of beneficial effects on the course of the disease and comorbidities that result from the downstream effects of seizures, whenever these beneficial effects can be explained solely by the suppression of seizure activity. It is acknowledged that other treatments, mostly under development, can exert direct favorable actions on the underlying disease or its progression, by having "antiepileptogenic" or "disease-modifying" effects. A more-refined terminology to describe precisely these actions needs to be developed.


Subject(s)
Epilepsy , Humans , Epilepsy/drug therapy , Epilepsy/etiology , Anticonvulsants/therapeutic use , Behavior Therapy , Consensus , Caregivers
7.
Transl Vis Sci Technol ; 9(8): 30, 2020 07.
Article in English | MEDLINE | ID: mdl-32855876

ABSTRACT

The Foundation Fighting Blindness, RDH12 family organizations, and the RDH12 Fund for Sight convened a jointly organized workshop in Columbia, Maryland, on November 19, 2019. The purpose of the workshop was to share perspectives on what is known about the RDH12-associated retinal dystrophies (RDs) and discuss the advancement of therapies, primarily gene therapy, for people with mutations in the RDH12 gene which cause Leber congenital amaurosis 13 (LCA13). The workshop began with presentations on the RDH12 landscape, patient perspectives, the use of statistical modeling for clinical trial design, and the Foundation's My Retina Tracker Registry. An afternoon roundtable discussion focused on four key areas essential to advance research toward gene therapy clinical trials: trial design, dose projection from nonclinical to clinical studies, natural history, and regulatory considerations. In their comments, the 27 participants from academic centers, affected families, biotechnology and pharmaceutical companies, and the regulatory community highlighted a number of research priorities, including the following: systematic inventory of retrospective natural history studies and planning for a multicenter prospective study, development of large animal models, and evaluation of novel tests of functional vision in young children. Despite these research opportunities, the workshop participants agreed that the field could be ready now for a clinical trial aimed initially at testing the safety and, possibly, efficacy of RDH12 gene therapy. Advancements in this field are being fostered by the emergence of an innovative multi-stakeholder research endeavor that relies on the effective engagement of the patients. Translational Relevance: This initiative serves as a model for how affected individuals and their families can be research partners on the path to treatments and cures.


Subject(s)
Alcohol Oxidoreductases , Retinal Dystrophies , Alcohol Oxidoreductases/genetics , Animals , Child , Child, Preschool , Humans , Maryland , Mutation , Prospective Studies , Retrospective Studies
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