{"id":1684,"date":"2012-09-24T22:30:31","date_gmt":"2012-09-24T22:30:31","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?page_id=1684"},"modified":"2023-09-21T12:18:41","modified_gmt":"2023-09-21T16:18:41","slug":"first-ever-progeria-treatment","status":"publish","type":"page","link":"https:\/\/www.progeriaresearch.org\/es\/first-ever-progeria-treatment\/","title":{"rendered":"El primer tratamiento para la progeria"},"content":{"rendered":"<p>[et_pb_section fb_built=\u201d1\u2033 _builder_version=\u201d4.16\u2033 global_colors_info=\u201d{}\u201d][et_pb_row _builder_version=\u201d4.16\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201drepeat\u201d global_colors_info=\u201d{}\u201d][et_pb_column type=\u201d4_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text _builder_version=\u201d4.16\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201drepeat\u201d global_colors_info=\u201d{}\u201d]<\/p>\n<p>Se ha hecho historia: todos los ni\u00f1os que participaron en el primer ensayo cl\u00ednico con un medicamento para la progeria mostraron una mejor\u00eda en una o m\u00e1s \u00e1reas de su afecci\u00f3n, lo que demuestra que el medicamento lonafarnib de FTI es el primer tratamiento conocido y eficaz para ni\u00f1os con progeria.<\/p>\n<p><a href=\"https:\/\/www.pnas.org\/content\/109\/41\/16666\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>Haga clic aqu\u00ed para obtener una copia gratuita del documento con los resultados del ensayo cl\u00ednico<\/strong><\/a><\/p>\n<div style=\"width: 310px\" class=\"wp-caption alignleft\"><img loading=\"lazy\" decoding=\"async\" src=\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/photo.jpg\" alt=\"\" width=\"300\" height=\"205\" \/><p class=\"wp-caption-text\"><span style=\"font-size: 8pt;\"><strong>Megan y Meghan fueron las dos primeras ni\u00f1as que se inscribieron en el ensayo. Las dos ni\u00f1as se reunieron en Boston cada cuatro meses durante dos a\u00f1os para realizar pruebas, recibir nuevos medicamentos y jugar juntas. Aqu\u00ed est\u00e1n en diciembre de 2008, en un descanso de las pruebas en el Hospital Infantil de Boston.<\/strong><\/span><\/p><\/div>\n<p><strong>PARA LOS MEDIOS:\u00a0 <\/strong><a href=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/FINAL-PNAS-Press-Release.pdf\" target=\"_blank\" rel=\"noopener noreferrer\">haga clic aqu\u00ed<\/a> para el comunicado de prensa, b-roll y otros detalles de prensa.<\/p>\n<p><strong>Los resultados de <\/strong><a href=\"https:\/\/www.progeriaresearch.org\/es\/clinical-trials\/\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>El primer ensayo cl\u00ednico de un f\u00e1rmaco para ni\u00f1os<\/strong><\/a><strong> \u00a1Con Progeria ya estamos aqu\u00ed y es oficial!<\/strong>\u00a0 El lonafarnib, un tipo de inhibidor de la farnesiltransferasa (FTI) desarrollado originalmente para tratar el c\u00e1ncer, ha demostrado ser eficaz para la progeria. Todos los ni\u00f1os mostraron una mejor\u00eda en una o m\u00e1s de cuatro aspectos: aumento de peso, mejor audici\u00f3n, mejor estructura \u00f3sea y\/o, lo m\u00e1s importante, mayor flexibilidad de los vasos sangu\u00edneos. Los resultados del estudio, que se public\u00f3 en la revista <strong>Financiado y coordinado por la Fundaci\u00f3n para la Investigaci\u00f3n de la Progeria<\/strong>, se publicaron el 24 de septiembre de 2012 en <strong><em><a href=\"https:\/\/www.pnas.org\/content\/109\/41\/16666\" target=\"_blank\" rel=\"noopener noreferrer\">Actas de la Academia Nacional de Ciencias<\/a>.<\/em><\/strong><\/p>\n<p><em>Gordon et. al., Ensayo cl\u00ednico de un inhibidor de la farnesiltransferasa en ni\u00f1os con s\u00edndrome de progeria de Hutchinson-Gilford, PNAS, 9 de octubre de 2012 vol. 109 no. 41 16666-16671<\/em><\/p>\n<div style=\"width: 297px\" class=\"wp-caption alignright\"><img loading=\"lazy\" decoding=\"async\" class=\"alignright\" src=\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/Natsuki-family-drug-prep.jpg\" alt=\"\" width=\"287\" height=\"210\" \/><p class=\"wp-caption-text\"><span style=\"font-size: 8pt;\"><strong>Natsuki, de Jap\u00f3n, con su hermano, su padre y su madre quienes preparan la mezcla de lonafarnib y edulcorante l\u00edquido.<\/strong><\/span><\/p><\/div>\n<p style=\"text-align: left;\"><strong>Los resultados muestran mejoras en todos los ni\u00f1os<\/strong><br \/>Veintiocho ni\u00f1os de diecis\u00e9is pa\u00edses participaron en el ensayo de dos a\u00f1os y medio de duraci\u00f3n, lo que representa el 75 por ciento de los casos conocidos de progeria en todo el mundo en el momento en que comenz\u00f3 el ensayo. Los ni\u00f1os viajaban a Boston cada cuatro meses para recibir pruebas m\u00e9dicas exhaustivas y estudiar los medicamentos a trav\u00e9s del Boston Children&#039;s Hospital. <a href=\"https:\/\/www.childrenshospital.org\/research\/clinical-research\/icctr\" target=\"_blank\" rel=\"noopener noreferrer\">Unidad de Estudios Cl\u00ednicos y Traslacionales<\/a>Todos recibieron lonafarnib oral, un f\u00e1rmaco inyectable suministrado por Merck &amp; Co., dos veces al d\u00eda durante dos a\u00f1os, bajo la supervisi\u00f3n del presidente del ensayo cl\u00ednico, el Dr. Mark Kieran, Ph.D., Director de Neurooncolog\u00eda M\u00e9dica Pedi\u00e1trica en el Centro Oncol\u00f3gico del Hospital de Ni\u00f1os Dana-Farber, y los copresidentes, la Dra. Monica Kleinman y la Dra. Leslie Gordon.<\/p>\n<p style=\"text-align: left;\">El principal criterio de valoraci\u00f3n fue la tasa de aumento de peso, ya que los ni\u00f1os con progeria sufren un retraso grave del crecimiento, con una tasa de aumento de peso lineal muy lenta a lo largo del tiempo. Los investigadores examinaron muchas otras \u00e1reas del cuerpo, incluida la rigidez arterial (un predictor de ataque card\u00edaco y accidente cerebrovascular), la rigidez \u00f3sea (un indicador de la fortaleza \u00f3sea) y la audici\u00f3n. &quot;Cuando comenzamos este ensayo cl\u00ednico, no ten\u00edamos idea de si alg\u00fan aspecto de la progeria ser\u00eda reversible, porque nadie hab\u00eda realizado antes un ensayo de tratamiento cl\u00ednico para la progeria. Descubrimos que, entre otras cosas, los principales vasos sangu\u00edneos pueden mejorar realmente. Este fue un descubrimiento revolucionario para los ni\u00f1os, ya que la enfermedad cardiovascular acelerada es la causa de muerte en la progeria. Aunque no hay forma de saber si hemos retrasado los accidentes cerebrovasculares, los ataques card\u00edacos o aumentado la longevidad en un per\u00edodo de tratamiento de solo 2 a\u00f1os, estos resultados positivos nos obligan a seguir impulsando nuevos tratamientos hasta que logremos lo que nos propusimos hacer en 1999. Queremos que los ni\u00f1os con progeria vivan hasta los 80 a\u00f1os y m\u00e1s. \u201cQueremos que vivan vidas plenas y saludables\u201d, dijo el Dr. Gordon, director m\u00e9dico de PRF y primer autor del art\u00edculo sobre el descubrimiento del tratamiento.<\/p>\n<p><strong>Un ritmo r\u00e9cord de progreso<\/strong><\/p>\n<p style=\"text-align: justify;\">El descubrimiento del tratamiento se produce menos de una d\u00e9cada despu\u00e9s de que PRF y el actual director de los Institutos Nacionales de Salud, el Dr. Francis Collins, unieran sus fuerzas para identificar la causa de la progeria, \u00a1un cronograma inaudito en el mundo de la investigaci\u00f3n m\u00e9dica! Pero para PRF y los ni\u00f1os con progeria, que viven una edad promedio de solo 13 a\u00f1os, esa velocidad es vital para ganar esta carrera contra el tiempo.<\/p>\n<div style=\"width: 260px\" class=\"wp-caption alignleft\"><img loading=\"lazy\" decoding=\"async\" src=\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/mateoMilagorsFernando.jpg\" alt=\"\" width=\"250\" height=\"194\" \/><p class=\"wp-caption-text\"><span style=\"font-size: 8pt;\"><strong>Mateo, Milagros y Fernando sonr\u00eden al recibir los premios de la PRF en su \u00faltima visita. Los trofeos dicen: &quot;\u00a1LO LOGRASTE! Terminaste el primer ensayo sobre progeria. \u00a1Eres una SUPERESTRELLA!&quot;<\/strong><\/span><\/p><\/div>\n<p>\u201cPRF es un buen ejemplo de una organizaci\u00f3n que ha permitido con \u00e9xito la investigaci\u00f3n traslacional, pasando del descubrimiento de genes al tratamiento cl\u00ednico a un ritmo sin precedentes\u201d, afirm\u00f3 el Dr. Kieran. \u201cDesde 1999, cuando se fund\u00f3 la organizaci\u00f3n, hasta hoy, PRF ha identificado la mutaci\u00f3n gen\u00e9tica que causa la enfermedad, ha financiado la investigaci\u00f3n precl\u00ednica, ha completado este ensayo, ha iniciado un segundo ensayo y actualmente est\u00e1 trabajando con nuestro equipo en el Boston Children&#039;s Hospital para planificar otro ensayo con medicamentos que, como los FTI, han mostrado resultados emocionantes en c\u00e9lulas de progeria y modelos animales. Es un historial de logros impresionante.<\/p>\n<p><strong>\u00a1ESTAMOS DE ACUERDO! <\/strong><strong>\u00bfC\u00f3mo llegamos a este maravilloso d\u00eda?<\/strong><br \/>Despu\u00e9s de 2003 <a href=\"https:\/\/www.progeriaresearch.org\/es\/2003\/04\/16\/progeria-gene-discovered\/\">Descubrimiento de la mutaci\u00f3n gen\u00e9tica que causa la progeria<\/a>, Investigadores financiados por el PRF identificados <a href=\"https:\/\/www.progeriaresearch.org\/es\/assets\/files\/pdf\/FTIQ&amp;AAugust2006Final.pdf\/\">FTI<\/a> como posible tratamiento farmacol\u00f3gico. La mutaci\u00f3n que causa la progeria conduce a la producci\u00f3n de la prote\u00edna <em>progerina<\/em>, que da\u00f1a la funci\u00f3n celular. Parte del efecto t\u00f3xico de la progerina en el cuerpo es causado por una mol\u00e9cula llamada &quot;grupo farnesilo&quot;, que se adhiere a la prote\u00edna progerina y la ayuda a da\u00f1ar las c\u00e9lulas del cuerpo. Los FTI act\u00faan bloqueando la uni\u00f3n del grupo farnesilo a la progerina, lo que reduce el da\u00f1o que causa la progerina.<\/p>\n<p><strong>Para m\u00e1s detalles del estudio, <\/strong><a href=\"https:\/\/www.progeriaresearch.org\/es\/assets\/files\/pdf\/FINAL%20PNAS%20Press%20Release.pdf\/\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>HAGA CLIC AQU\u00cd para ver el comunicado de prensa<\/strong><\/a><\/p>\n<p><strong>La progeria est\u00e1 relacionada con el proceso normal de envejecimiento<\/strong><a href=\"https:\/\/www.progeriaresearch.org\/es\/whats-new-in-progeria-research\/\" target=\"_blank\" rel=\"noopener noreferrer\"><br \/>Investigaci\u00f3n<\/a> muestra que la prote\u00edna causante de la progeria <em>progerina<\/em> Tambi\u00e9n se produce en la poblaci\u00f3n general y aumenta con la edad. Los investigadores planean seguir explorando el efecto de los FTI, que pueden ayudar a los cient\u00edficos a aprender m\u00e1s sobre la enfermedad cardiovascular que afecta a millones de personas, as\u00ed como el proceso normal de envejecimiento que nos afecta a todos.<\/p>\n<blockquote>\n<p>\u201cLa conexi\u00f3n entre esta enfermedad rara y el envejecimiento normal est\u00e1 dando frutos de manera importante... se obtienen valiosos conocimientos biol\u00f3gicos mediante el estudio de trastornos raros como la progeria. Nuestra sensaci\u00f3n desde el principio fue que la progeria ten\u00eda mucho que ense\u00f1arnos sobre el proceso normal de envejecimiento\u201d.<\/p>\n<p>\u2013 Dr. Francis Collins, Director de los Institutos Nacionales de Salud<\/p>\n<\/blockquote>\n<div style=\"width: 360px\" class=\"wp-caption alignright\"><img loading=\"lazy\" decoding=\"async\" src=\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/Maria-Painting.jpg\" alt=\"\" width=\"350\" height=\"189\" \/><p class=\"wp-caption-text\"><span style=\"font-size: 8pt;\"><strong>\u00a1Qu\u00e9 sonrisa! A Mar\u00eda le gust\u00f3 mucho pintar durante uno de sus descansos de las pruebas en el Boston Children&#039;s Hospital.<\/strong><\/span><\/p><\/div>\n<p><strong>Ay\u00fadenos a encontrar a todos los ni\u00f1os con progeria para que ellos tambi\u00e9n puedan beneficiarse de nuestro trabajo<\/strong><br \/>Los investigadores creen que en un momento dado, hay entre 200 y 250 ni\u00f1os que viven con progeria. Para identificar a los ni\u00f1os desconocidos, PRF lanz\u00f3 el proyecto <a href=\"https:\/\/www.progeriaresearch.org\/es\/find-the-children\/\" target=\"_blank\" rel=\"noopener noreferrer\">\u201cEncuentra los otros 150\u201d<\/a> Campa\u00f1a de sensibilizaci\u00f3n en octubre de 2009 y, a partir de septiembre de 2012, sabemos de 96 ni\u00f1os que viven en 35 pa\u00edses, \u00a1un aumento de 83%! Usted puede ayudar a encontrar m\u00e1s ni\u00f1os para que puedan beneficiarse del tratamiento y la atenci\u00f3n exclusivos que brinda PRF. <em>Estos nuevos ni\u00f1os pueden ser elegibles para futuros ensayos cl\u00ednicos, as\u00ed que por favor visite <\/em><a href=\"https:\/\/www.progeriaresearch.org\/es\/find-the-children\/\" target=\"_blank\" rel=\"noopener noreferrer\"><em>Encuentra los otros 150<\/em><\/a><em>\u00a0para descubrir c\u00f3mo puedes ayudar a que esto suceda.<\/em><\/p>\n<p><a href=\"https:\/\/www.progeriaresearch.org\/es\/2012\/05\/16\/a-special-thank-you\/\"><strong>Gracias a todos <\/strong><strong>\u2013<\/strong> <strong>\u00a1No podr\u00edamos haberlo hecho sin ti!<\/strong><\/a><br \/>Una de las principales razones por las que logramos resultados revolucionarios en este primer ensayo es gracias a los tremendos patrocinadores que nos brindaron financiaci\u00f3n y otro tipo de apoyo, ayud\u00e1ndonos a acercarnos un paso m\u00e1s a nuestro objetivo final: una cura para la progeria. Haga clic aqu\u00ed para ver un homenaje especial a todos aquellos que ayudaron a que el ensayo <em>sue\u00f1o<\/em> de un tratamiento a <strong><em>realidad<\/em><\/strong>.<\/p>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":"<p>[et_pb_section fb_built=\u201d1\u2033 _builder_version=\u201d4.16\u2033 global_colors_info=\u201d{}\u201d][et_pb_row _builder_version=\u201d4.16\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201drepeat\u201d global_colors_info=\u201d{}\u201d][et_pb_column type=\u201d4_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text _builder_version=\u201d4.16\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201drepeat\u201d global_colors_info=\u201d{}\u201d] Se ha hecho historia: todos los ni\u00f1os que participaron en el primer ensayo cl\u00ednico con medicamentos para la progeria mostraron una mejor\u00eda en una o m\u00e1s \u00e1reas de su afecci\u00f3n, lo que demuestra que el medicamento FTI, lonafarnib, es el primer tratamiento conocido y eficaz [\u2026]<\/p>","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"<p>History has been made, with every child in the first-ever Progeria clinical drug trial showing improvement in one or more areas of their condition, proving that the FTI drug lonafarnib is the first known, effective treatment for children with Progeria.<\/p><p><a href=\"https:\/\/web-beta.archive.org\/web\/20160518205005\/https:\/\/www.pnas.org\/content\/109\/41\/16666\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>Click here for your free copy of the clinical trial results paper<\/strong><\/a><\/p>[caption id=\"\" align=\"alignleft\" width=\"300\"]<img src=\"https:\/\/web-beta.archive.org\/web\/20170216095030im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/photo.jpg\" alt=\"\" width=\"300\" height=\"205\" \/> <span style=\"font-size: 8pt;\"><strong>Megan and Meghan were the first two children to enroll in the trial. The two girls met in Boston every four months for two years, for testing, to receive new drug supply, and to play together! Here they are in December 2008, on a break from tests at Boston Children\u2019s\u2019 Hospital.<\/strong><\/span>[\/caption]<p><strong>FOR MEDIA:\u00a0 <\/strong><a href=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/FINAL-PNAS-Press-Release.pdf\" target=\"_blank\" rel=\"noopener noreferrer\">click here<\/a> for the press release, b-roll and other press details.<\/p><p><strong>The results of <\/strong><a href=\"https:\/\/www.progeriaresearch.org\/clinical-trials\/\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>the first-ever clinical drug trial for children<\/strong><\/a><strong> with Progeria are in and it\u2019s official!<\/strong>\u00a0 Lonafarnib, a type of farnesyltransferase inhibitor (FTI) originally developed to treat cancer, has proven effective for Progeria. Every child showing improvement in one or more of four ways: gaining additional weight, better hearing, improved bone structure and\/or, most importantly, increased flexibility of blood vessels. Results of the study, which was <strong>funded and coordinated by The Progeria Research Foundation<\/strong>, were published September 24, 2012 in <strong><em><a href=\"https:\/\/web-beta.archive.org\/web\/20160518205005\/https:\/\/www.pnas.org\/content\/109\/41\/16666\" target=\"_blank\" rel=\"noopener noreferrer\">Proceedings of the National Academy of Sciences<\/a>.<\/em><\/strong><\/p><p><em>Gordon et. al., Clinical Trial of a Farnesyltransferase Inhibitor in Children with Hutchinson-Gilford Progeria Syndrome, PNAS, October 9, 2012 vol. 109 no. 41 16666-16671<\/em><\/p>[caption id=\"\" align=\"alignleft\" width=\"287\"]<img class=\"alignleft\" src=\"https:\/\/web-beta.archive.org\/web\/20170216095030im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/Natsuki-family-drug-prep.jpg\" alt=\"\" width=\"287\" height=\"210\" \/> <span style=\"font-size: 8pt;\"><strong>Natsuki, from Japan, with her brother, father and mother who prepares the lonafarnib-liquid sweetener mixture.<\/strong><\/span>[\/caption]<p style=\"text-align: left;\"><strong>Results Yield Improvements in All Children<\/strong><br \/>Twenty-eight children from sixteen countries participated in the 2 \u00bd year drug trial, representing 75 percent of known Progeria cases worldwide at the time the trial began. The children traveled to Boston every four months to receive comprehensive medical testing and study medications through Boston Children\u2019s Hospital\u2019s <a href=\"https:\/\/web-beta.archive.org\/web\/20170216095030\/https:\/\/www.childrenshospital.org\/cfapps\/research\/data_admin\/Site2734\/mainpageS2734P103.html\" target=\"_blank\" rel=\"noopener noreferrer\">Clinical and Translational Study Unit<\/a>. All received oral lonafarnib, an FTI supplied by Merck & Co., twice-a-day for two years, under the supervision of clinical trial chair <a href=\"https:\/\/www.childrenshospital.org\/researchers\/mark-kieran\" target=\"_blank\" rel=\"noopener noreferrer\">Mark Kieran, M.D., Ph.D.<\/a>, Director of Pediatric Medical Neuro-oncology at the Dana-Farber \/ Children\u2019s Hospital Cancer Center, and co-chairs <a href=\"https:\/\/web-beta.archive.org\/web\/20170216095030\/https:\/\/www.progeriaresearch.org\/volunteer-board.html#MKleinman\">Dr. Monica Kleinman<\/a> and <a href=\"https:\/\/web-beta.archive.org\/web\/20170216095030\/https:\/\/www.progeriaresearch.org\/officersandstaff.html\">Dr. Leslie Gordon<\/a><\/p><p style=\"text-align: left;\">Rate of weight gain was the primary outcome measure, because children with Progeria experience severe failure to thrive, with a very slow linear rate of weight gain over time. The researchers examined many other areas of the body, including arterial stiffness (a predictor of heart attack and stroke), bone rigidity (an indicator of bone strength) and hearing. \u201cWhen we started this clinical trial we had no idea whether any aspect of Progeria would be reversible, because no one had ever conducted a clinical treatment trial for Progeria before.\u00a0 We discovered that, among other things, the major blood vessels can actually improve.\u00a0 This was a breakthrough discovery for the children, since accelerated cardiovascular disease is the cause of death in Progeria. Though there is no way to know whether we have delayed strokes, heart attacks, or increased longevity within just a 2-year treatment period, these positive results compel us to continue pushing for new treatments until we accomplish what we set out to do in 1999. We want children with Progeria to live until they\u2019re 80 and beyond. We want them to live full, healthy lives,\u201d said Dr. Gordon, PRF\u2019s Medical Director and first author of the treatment discovery paper.<\/p><p><strong>Record Pace of Progress<\/strong><\/p><p style=\"text-align: justify;\">The treatment discovery comes less than a decade after PRF and now National Institutes of Health Director Dr. Francis Collins joined forces to identify the cause of Progeria \u2013 an unheard of timeline in the world of medical research!\u00a0 But for PRF and children with Progeria, who live to an average age of just 13 years, such speed is vital to win this race against time.<\/p>[caption id=\"\" align=\"alignleft\" width=\"250\"]<img src=\"https:\/\/web-beta.archive.org\/web\/20170216095030im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/mateoMilagorsFernando.jpg\" alt=\"\" width=\"250\" height=\"194\" \/> <span style=\"font-size: 8pt;\"><strong>Mateo, Milagros and Fernando are all smiles upon receiving awards from PRF at their final visit. The trophies say, \u201cYOU DID IT! You finished the 1st Progeria Trial \u2013 You\u2019re A SUPERSTAR!\u201d<\/strong><\/span>[\/caption]<p>\u201cPRF is a good example of an organization successfully enabling translational research, moving from gene discovery to clinical treatment at an unprecedented pace,\u201d said Dr. Kieran. \u201cFrom 1999, when the organization was founded, to today, PRF has identified the genetic mutation that causes the disease, funded preclinical research, completed this trial, initiated a second trial, and is currently working with our team at Boston Children\u2019s Hospital to plan yet another trial with drugs that, like FTIs, have shown exciting results in Progeria cells and animal models. That\u2019s an awesome track record of accomplishment.\u201d <strong><br \/>WE AGREE!<\/strong><strong>How Did We Get to This Wonderful Day?<\/strong><br \/>Following the 2003 <a href=\"https:\/\/web-beta.archive.org\/web\/20170216095030\/https:\/\/www.progeriaresearch.org\/progeria_gene_discovered.html\">discovery of the gene mutation that causes Progeria<\/a>, PRF-funded researchers identified <a href=\"https:\/\/web-beta.archive.org\/web\/20170216095030\/https:\/\/www.progeriaresearch.org\/assets\/files\/pdf\/FTIQ&AAugust2006Final.pdf\">FTIs<\/a> as a potential drug treatment. The Progeria-causing mutation leads to the production of the protein <em>progerin<\/em>, which damages cell function. Part of progerin\u2019s toxic effect on the body is caused by a molecule called a \u201cfarnesyl group,\u201d which attaches to the progerin protein and helps it damage the body\u2019s cells. FTIs act by blocking the attachment of the farnesyl group onto progerin, reducing the harm progerin causes.<\/p><p><strong>For more study details, <\/strong><a href=\"https:\/\/web-beta.archive.org\/web\/20170216095030\/https:\/\/www.progeriaresearch.org\/assets\/files\/pdf\/FINAL%20PNAS%20Press%20Release.pdf\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>CLICK HERE for the press release<\/strong><\/a><\/p><p><strong>Progeria Linked to Normal Aging Process<\/strong><a href=\"https:\/\/web-beta.archive.org\/web\/20170128050324\/https:\/\/www.progeriaresearch.org\/whats_new_in_progeria_research.html#2\" target=\"_blank\" rel=\"noopener noreferrer\"><br \/>Research<\/a> shows that the Progeria-causing protein <em>progerin<\/em> is also produced in the general population and increases with age. Researchers plan to continue exploring the effect of FTIs, which may help scientists learn more about the cardiovascular disease that affects millions, as well as the normal aging process that affects us all.<\/p><blockquote><p>\"Connecting this rare disease and normal aging is bearing fruit in an important way...valuable biological insights are gained by studying rare disorders such as Progeria. Our sense from the start was that Progeria had a lot to teach us about the normal aging process.\"<\/p><p>- Dr. Francis Collins, Director of the National Institutes of Health<\/p><\/blockquote>[caption id=\"\" align=\"alignright\" width=\"350\"]<img src=\"https:\/\/web-beta.archive.org\/web\/20170216095030im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/Maria-Painting.jpg\" alt=\"\" width=\"350\" height=\"189\" \/> <span style=\"font-size: 8pt;\"><strong>What a smile! Maria really enjoyed painting during one of her breaks from testing at Boston Children\u2019s Hospital.<\/strong><\/span>[\/caption]<p><strong>Help Us Find All Children With Progeria so They, Too, Can Benefit From Our Work<\/strong><br \/>Researchers believe that at any given time, there are 200-250 children living with Progeria. To identify unknown children, PRF launched the <a href=\"https:\/\/www.progeriaresearch.org\/find-the-other-150\/\" target=\"_blank\" rel=\"noopener noreferrer\">\u201cFind the Other 150\u201d<\/a> campaign in October 2009,\u00a0and as of September 2012, we know of 96 children living in 35 countries- an 83% increase!! You can help find more so they can benefit from the unique treatment and care that PRF provides. <em>These new children may be eligible for future clinical trials, so please go to <\/em><a href=\"https:\/\/www.progeriaresearch.org\/find-the-other-150\/\" target=\"_blank\" rel=\"noopener noreferrer\"><em>Find the other 150<\/em><\/a><em>\u00a0to find out how you can help make that happen.<\/em><\/p><p><span style=\"font-size: 10pt;\"><strong>What a smile! Maria really enjoyed painting during one<br \/>of her breaks from testing at Boston Children\u2019s Hospital.<\/strong><\/span><\/p><p><a href=\"https:\/\/web-beta.archive.org\/web\/20170216095030\/https:\/\/www.progeriaresearch.org\/a-special-thank-you.html\"><strong>Thank you ALL <\/strong><strong>-<\/strong> <strong>We Couldn\u2019t Have Done it Without You!<\/strong><\/a><br \/>One of the main reasons we achieved breakthrough results in this first trial is because of the tremendous supporters who provided funding and other support, helping to get us one step closer to achieving our ultimate goal \u2013 a cure for Progeria. Click here to see a special tribute to all those who helped make the <em>dream<\/em> of a treatment a <strong><em>reality<\/em><\/strong>.<\/p>","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"class_list":["post-1684","page","type-page","status-publish","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>treatment | The Progeria Research Foundation<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.progeriaresearch.org\/es\/first-ever-progeria-treatment\/\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"treatment | The Progeria Research Foundation\" \/>\n<meta property=\"og:description\" content=\"[et_pb_section fb_built=&#8221;1&#8243; _builder_version=&#8221;4.16&#8243; global_colors_info=&#8221;{}&#8221;][et_pb_row _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.16&#8243; custom_padding=&#8221;|||&#8221; global_colors_info=&#8221;{}&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.16&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221; global_colors_info=&#8221;{}&#8221;] History has been made, with every child in the first-ever Progeria clinical drug trial showing improvement in one or more areas of their condition, proving that the FTI drug lonafarnib is the first known, effective treatment [&hellip;]\" \/>\n<meta property=\"og:url\" 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