The Niemann-Pick Spain Foundation has given IDIBELL and Bellvitge Hospital
\na cheque for 104,884 euros that will be used entirely for the continuation of a
\nstudy to determine the efficacy and safety of the drug Efavirenz in the treatment
\nof cognitive impairment in patients with this rare genetic disease for which there
\nis currently no cure. The first results of the trial have provided quite encouraging
\nresults.
\nThe president of the foundation, Carmelo Fern\u00e1ndez, gave the cheque to Dr.
\nJordi Gascon, neurologist and principal investigator of the neurological and
\nneurogenic diseases group of IDIBELL and Bellvitge Hospital, in an event at the
\ncenter, on November 8. Dr. Gabriel Capell\u00e0, director of the Institute, and S\u00edlvia
\nCordom\u00ed, deputy manager of the HUB and the Viladecans Hospital, as well as
\nother members of the Niemann-Pick Foundation, also attended.
\nDr. Gasc\u00f3n thanked the tireless effort of the Niemann-Pick Foundation to
\npromote research as a tool to improve the quality of life of patients with an
\nindependent clinical trial carried out with all the guarantees. Carried out with 16
\npatients (there are about 27 in the whole of Spain), the first results of the
\nadministration of Efavirenz indicate benefits in cognitive and behavioral scales,
\nespecially in patients with less time of evolution of the disease. That is why new
\nstudies are now needed to expand the results, as Dr. Gasc\u00f3n highlighted.
\nEfavirenz is an antiretroviral drug used in the treatment of HIV that improves the
\nfunction of an enzyme that regulates the distribution of cholesterol at the
\nsynapse, making it a great candidate for Niemann-Pick.
\nNiemann-Pick disease is a hereditary neurodegenerative disease characterized
\nby the accumulation of different types of lipids (sphingomyelin, cholesterol) that
\ncauses visceral and nervous system alterations. It is divided into four types (A,
\nB, C, and D) depending on the affected gene, which determines a different
\nclinical profile. The most common symptoms are cognitive impairment, epilepsy,
\nbehavioural disorder, vertical gaze paralysis, ataxia, dystonia and
\nsplenomegaly. Existing treatments are discreetly effective, but they slow down
\nthe evolution of the disease.<\/p>\n
<\/p>\n
The Bellvitge Biomedical Research Institute (IDIBELL) is a biomedical research center created in 2004. It is participated by the Bellvitge University Hospital and the Viladecans Hospital of the Catalan Institute of Health, the Catalan Institute of Oncology, the University of Barcelona and the City Council of L\u2019Hospitalet de Llobregat.<\/em><\/p>\n IDIBELL is a member of the Campus of International Excellence of the University of Barcelona HUBc and is part of the CERCA institution of the Generalitat de Catalunya. In 2009 it became one of the first five Spanish research centers accredited as a health research institute by the Carlos III Health Institute. In addition, it is part of the \u201cHR Excellence in Research\u201d program of the European Union and is a member of EATRIS and REGIC. Since 2018, IDIBELL has been an Accredited Center of the AECC Scientific Foundation (FCAECC).<\/em><\/p>\n","protected":false},"excerpt":{"rendered":" Dr. Gasc\u00f3n thanked the tireless effort of the Niemann-Pick Foundation to
\npromote research as a tool to improve patients' quality of life.
\nEfavirenz is an antiretroviral drug used in HIV treatment that improves the
\nfunction of an enzyme that regulates cholesterol.<\/p>\n","protected":false},"author":8,"featured_media":24790,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","theme-transparent-header-meta":"default","adv-header-id-meta":"","stick-header-meta":"default","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"set","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"categories":[239,301,188],"tags":[],"class_list":["post-24789","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-neurologic-diseases-and-neurogenetics","category-neuroscience-area","category-neuroscience-program"],"publishpress_future_action":{"enabled":false,"date":"2024-12-29 03:03:00","action":"change-status","newStatus":"draft","terms":[],"taxonomy":"category"},"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/posts\/24789","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/users\/8"}],"replies":[{"embeddable":true,"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/comments?post=24789"}],"version-history":[{"count":1,"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/posts\/24789\/revisions"}],"predecessor-version":[{"id":24791,"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/posts\/24789\/revisions\/24791"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/media\/24790"}],"wp:attachment":[{"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/media?parent=24789"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/categories?post=24789"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/idibell.cat\/en\/wp-json\/wp\/v2\/tags?post=24789"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}