Click Here<\/strong>\u00a0for more details.<\/a><\/p>\n Association of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome, Leslie B. Gordon, MD, PhD; Heather Shappell, PhD; Joe Massaro, PhD; Ralph B. D\u2019Agostino Sr., PhD; Joan Brazier, MS; Susan E. Campbell, MA; Monica E. Kleinman, MD; Mark W. Kieran, MD, PhD;\u00a0JAMA,<\/em>\u00a0April 24, 2018.<\/p>\n<\/div>\n<\/div>\n<\/div>\n [\/et_pb_toggle][et_pb_toggle title=”July 2016: Triple Trial Results” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”July 2016: Triple Trial Results” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” hover_enabled=”0″ z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n July 2016: Triple Trial Results<\/a><\/p>\n<\/div>\n [\/et_pb_toggle][et_pb_toggle title=”October 2014: PRF\u2019s remarkable journey published in Expert Opinion” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”October 2014: PRF\u2019s remarkable journey published in Expert Opinion” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}”]<\/p>\n *\u201dThe Progeria Research Foundation: its remarkable journey from obscurity to treatment\u201d October 30, 2014<\/a><\/p>\n<\/div>\n<\/div>\n The authors write, \u201cIt is our hope that the description of the PRF programs and services that follows, along with an account of how they are helping PRF accomplish its mission to save children with Progeria, will assist and inspire others to take similar action for the many rare disease populations that need immediate attention.\u201d<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n [\/et_pb_toggle][et_pb_toggle title=”May 2014: Study Finds Trial Medications Increase Estimated Lifespan in Children With Progeria” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”May 2014: Study Finds Trial Medications Increase Estimated Lifespan in Children With Progeria” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}”]<\/p>\n This study demonstrates there is evidence that a farnesyltransferase inhibitor (FTI) can extend the lives of children with Progeria by at least one-and-a-half years. The study showed an extension of mean survival of 1.6 years during the six years following initiation of treatment. Two additional drugs added later in the trials, pravastatin and zoledronate, may also contribute to this finding.\u00a0This is the first evidence of treatments influencing survival for this fatal disease.<\/strong><\/p>\n Click here<\/a>\u00a0for more details.<\/p>\n Impact of Farnesylation Inhibitors on Survival in Hutchinson-Gilford Progeria Syndrome, Leslie B. Gordon, MD, PhD, Joe Massaro, PhD, Ralph B. D\u2019Agostino Sr., PhD, Susan E. Campbell, MA, Joan Brazier, MS, W. Ted Brown, MD, PhD, Monica E Kleinman, MD, Mark W. Kieran MD, PhD and the Progeria Clinical Trials Collaborative;\u00a0Circulation<\/em>, May 2, 2014 (on-line).<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n [\/et_pb_toggle][et_pb_toggle title=”September 2012: First-ever Progeria Treatment for Progeria Discovered” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”September 2012: First-ever Progeria Treatment for Progeria Discovered” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” hover_enabled=”0″ z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n The results of\u00a0the first-ever clinical drug trial for children<\/a>\u00a0with Progeria reveal that\u00a0Lonafarnib, 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. The study* was\u00a0funded and coordinated by The Progeria Research Foundation.<\/p>\n Click here<\/a>\u00a0for more details.<\/p>\n *Gordon LB<\/strong>, Kleinman ME, Miller DT, Neuberg D, Giobbie-Hurder A, Gerhard-Herman M, Smoot L, Gordon CM, Cleveland R, Snyder BD, Fligor B, Bishop WR, Statkevich P, Regen A, Sonis A, Riley S, Ploski C, Correia A, Quinn N, Ullrich NJ, Nazarian A, Liang MG, Huh SY, Schwartzman A, Kieran MW, Clinical Trial of a Farnesyltransferase Inhibitor in Children with Hutchinson-Gilford Progeria Syndrome,\u00a0Proceedings of the National Academy of Sciences<\/a>,\u00a0<\/strong>October 9, 2012\u00a0vol. 109\u00a0no. 41\u00a016666-16671<\/p>\n [\/et_pb_toggle][et_pb_toggle title=”October 2011: A Novel Approach to Progeria Therapy” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”October 2011: A Novel Approach to Progeria Therapy” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}”]<\/p>\n Spanish and French scientists under the leadership of Carlos L\u00f3pez-Otin (Oviedo) and Nicolas L\u00e9vy (Marseille) have published an exciting study that may result in a new approach to treating Progeria (1). While drugs used in PRF\u2019s clinical trials to date have targeted changes made in the abnormal lamin A protein (progerin) that is made in Progeria cells, \u00a0in the new work, the aberrant \u201csplicing\u201d of the lamin A messenger RNA (mRNA) coding for the lamin A protein is blocked, resulting in lowering the production of progerin. \u00a0 The blocking agent used is a small modified RNA molecule whose sequence is complementary to the region of the Progeria mRNA at which the splicing occurs. \u00a0This molecule binds to the splice site and prevents the binding there of the complex of protein and RNA molecules required for splicing (the \u201cspliceosome\u201d).<\/p>\n<\/div>\n That aberrant splicing in cultured skin cells of Progeria can be prevented in this manner was shown in \u00a02005 (2). \u00a0However, for treatment of patients the inhibiting reagent must be delivered intact to all tissues of the patient. \u00a0it took another six years, and work in several laboratories, to develop these \u201cdelivery\u201d methods.<\/p>\n In the new research (1), blocking the aberrant splicing in the model mouse resulted in impressive results. There were clear reductions in progerin concentrations in all tissues analyzed except skeletal muscle, which may have a reduced uptake of the blocking agent. \u00a0The model mice recapitulated many of the phenotypes of Progeria patients, including<\/p>\n The\u00a0in vivo\u00a0<\/strong>demonstration of the efficacy of reducing progerin production by blocking the aberrant splicing is a strong candidate for a valuable new approach to Progeria therapy.<\/p>\n (1) Osorio FG, Navarro CL, Cadi\u00f1anos J, L\u00f3pez-Mejia IC, Quir\u00f3s PM, et al, Science Translational Medicine,\u00a03:\u00a0<\/strong>Issue 106, advance on-line publication,\u00a0October 26 (2011).<\/p>\n (2) Scaffidi, P. and Misteli, T. Reversal of the , cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome, Nature Medicine\u00a011<\/strong>\u00a0(4): 440-445 (2005).<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n [\/et_pb_toggle][et_pb_toggle title=”June 2011: PRF-funded study Identifies Rapamycin as Possible Treatment for Progeria” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”June 2011: PRF-funded study Identifies Rapamycin as Possible Treatment for Progeria” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}”]<\/p>\n Researchers at the National Institutes of Health and Massachusetts General Hospital in Boston, MA published a new study today in\u00a0Science, Translational Medicine<\/em>\u00a0that may lead to a new drug treatment for children with Progeria.*<\/p>\n Rapamycin<\/em> is an FDA approved drug that has previously been shown to extend the lives of non-progeria mouse models. This new study demonstrates that rapamycin decreases the amount of the disease-causing protein progerin by 50%, improves the abnormal nuclear shape, and extends the lifespan of progeria cells. This study provides the first evidence that rapamycin may be able to decrease progerin\u2019s damaging effects in children with progeria.<\/p>\n There is tremendous media coverage on this! Click below for links to media stories:<\/p>\n Wall Street Journal Health Blog<\/a><\/strong><\/p>\n US News and World Report<\/a><\/strong><\/p>\n Science Magazine<\/a><\/strong><\/p>\n Boston Globe<\/a><\/strong><\/p>\n CNN<\/a> The Progeria Research Foundation was delighted to provide cells for this project from the\u00a0PRF Cell & Tissue Bank<\/a><\/strong>, and help fund the research through our\u00a0grants program<\/a>.<\/strong><\/p>\n This exciting new study demonstrates the remarkable pace of progeria research, while providing further insight into the aging process that affects us all.<\/p>\n *\u201dRapamycin Reverses Cellular Phenotypes and Enhances Mutant Protein Clearance in Hutchinson-Gilford Progeria Cells\u201d [\/et_pb_toggle][et_pb_toggle title=”June 2011: Groundbreaking Study on Progeria-Aging Link” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”June 2011: Groundbreaking Study on Progeria-Aging Link” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}”]<\/p>\n CBS Evening News<\/a>,\u00a0Wall Street Journal<\/a>\u00a0and\u00a0Others<\/a>\u00a0Report on New Study<\/p>\n National Institutes of Health researchers have discovered a previously unknown link between Progeria and aging.\u00a0 The findings provide insights about the relationship between the toxic, Progeria-causing protein known as\u00a0progerin\u00a0<\/strong>and\u00a0telomeres<\/strong>, which protect the ends of DNA within cells until they wear away over time and the cells die.<\/p>\n Progerin-expressing cells from normal individuals show signs of senescence.DNA in the nucleus is stained blue. Telomeres are seen as red dots.<\/p><\/div>\n The study* appears in the June 13, 2011 early online edition of the Journal of Clinical Investigation. It concludes that in normal aging, short or dysfunctional telomeres stimulate cells to produce progerin, which is associated with age-related cell damage.<\/p>\n \u201cFor the first time, we know that telomere shortening and dysfunction influences the production of progerin,\u201d says The Progeria Research Foundation Medical Director Leslie B. Gordon, MD, PhD. \u201cThus these two processes, both of which influence cellular aging, are actually linked.\u201d<\/strong><\/p>\n Prior research has shown that progerin is not only produced in children with Progeria, but that it is produced in smaller amounts in all of us, and progerin levels increase with aging. Independently, previous research on telomere shortening and dysfunction has been associated with normal aging. Since 2003, with the discovery of the Progeria gene mutation and the progerin protein that causes the disease, one of the key areas of research has focused on understanding whether and how Progeria and aging are linked.<\/p>\n \u201cConnecting this rare disease phenomenon and normal aging is bearing fruit in an important way,\u201d said NIH Director Francis S. Collins, MD, PhD, a senior author of the paper. \u201cThis study highlights that valuable biological insights are gained by studying rare genetic disorders such as Progeria. Our sense from the start was that Progeria had a lot to teach us about the normal aging process. \u201c<\/p>\n Scientists have traditionally studied telomeres and progerin separately. While there is still much to learn about whether this new connection can lead to a cure for children with Progeria or potentially be applied to extending the human lifespan, this study provides further evidence that progerin, the toxic protein discovered through finding the gene mutation in Progeria, plays a role in the normal aging process.<\/p>\n![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/02\/Figure-1.jpg\")
In an article published in\u00a0Expert Opinion<\/em>\u00a0and authored by Executive Director Audrey Gordon and Medical Director Leslie Gordon, the two PRF leaders discuss PRF\u2019s history, goals and accomplishments, and how the PRF programs have been pivotal in the journey from obscurity to treatment.<\/p>\n\n
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Kan Cao, John J. Graziotto, Cecilia D. Blair, Joseph R. Mazzulli, Michael R. Erdos, Dimitri Krainc, Francis S. Collins<\/strong>
Sci Transl Med. 2011 Jun 29;3(89):89ra58.<\/strong><\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n![\"\"](\"https:\/\/www.progeriaresearch.org\/assets\/images\/medical_images\/panel.jpg\")
We\u2019re excited to share two thrilling research updates with you, published online today in the world\u2019s top cardiovascular journal, Circulation (1):<\/em><\/p>\n Biomarker in Progeria This test can optimize the clinical trial process by\u00a0providing early information about the effectiveness of treatments being tested<\/strong>, as a lead-in to other clinical tests such as weight gain, dermatologic changes, joint contracture and function, etc., all of which require much more time to manifest. These clinical features of Progeria are important longer-term measures of treatment effects that are now complemented by the progerin levels measured earlier in therapy. We may now be able to understand treatment benefits as early as four months after starting treatment, or stop a treatment that may not benefit the trial participant, to avoid unnecessary side effects.<\/p>\n Even longer lives with lonafarnib Study data indicate that lower progerin levels in the blood reflected survival benefit: the longer someone with Progeria stayed on lonafarnib, the greater the survival benefit from being on therapy. Progerin levels were decreased by about 30-60% for as long as the drug was taken, and life expectancy for patients on treatment for over 10 years was estimated to increase by almost 5 years. That\u2019s more than a 35% increase in average lifespan, from 14.5 years to almost 20 years of age<\/strong>!<\/p>\n To learn more, see our press release here<\/strong><\/a><\/p>\n \u201cOne of the most remarkable stories ever shared on this podcast\u201d And in June, two editorial papers\u00a0(2)\u00a0<\/a>and\u00a0(3)<\/a>\u00a0were published in\u00a0Circulation\u00a0<\/em>highlighting this biomarker\u2019s critical importance to advancing treatments and the cure for children with Progeria and for better understanding aging.<\/p>\n (1) Gordon, L.B., Norris, W., Hamren, S.,\u00a0et al<\/em>. Plasma Progerin in Patients with Hutchinson-Gilford Progeria Syndrome: Immunoassay Development and Clinical Evaluation.\u00a0Circulation<\/em>, 2023<\/p>\n (2)\u00a0Progression of Cardiac Abnormalities in Hutchinson-Gilford Progeria Syndrome: A Prospective Longitudinal Study.<\/a> (3)\u00a0Readily Available Tools to Detect Progerin and Cardiac Disease Progression in Hutchinson-Gilford Progeria Syndrome.<\/a> [\/et_pb_toggle][et_pb_toggle title=”March 2021: Exciting breakthroughs in RNA Therapeutics for Progeria!” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”March 2021: Exciting breakthroughs in RNA Therapeutics for Progeria!” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” hover_enabled=”0″ z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n We\u2019re thrilled to share the results from\u00a0two very exciting breakthrough studies on the use of RNA therapeutics<\/strong>\u00a0in Progeria research.\u00a0 Both studies were co-funded by The Progeria Research Foundation (PRF) and co-authored by PRF\u2019s Medical Director, Dr. Leslie Gordon.<\/p>\n Progerin is the disease-causing protein in Progeria. The RNA therapies interfere with the body\u2019s ability to produce progerin, by blocking its production at the RNA level.\u00a0 This means that\u00a0the treatment is more specific than most therapies<\/strong>\u00a0that target progerin at the protein level.<\/p>\n Though each study used a different drug delivery system, both studies targeted the same basic treatment strategy, inhibiting production of RNA coding for the abnormal protein, progerin. Both were led by researchers at the National Institutes of Health (NIH), and were published today in the journal\u00a0Nature Medicine<\/em>.<\/p>\n One study<\/a>, led by Francis Collins, MD, PhD, Director of the NIH, showed that treating Progeria mice with a drug named SRP2001 reduced the harmful progerin mRNA and protein expression in the aorta<\/strong>, the main artery in the body, as well as in other tissues. At the end of the study, the aortic wall remained stronger and the mice demonstrated an\u00a0increased survival of over 60%<\/strong>.<\/p>\n \u201cTo have a targeted RNA-therapy show such significant results in an animal model gives me hope that this could lead to a major advance for the treatment of progeria,\u201d said Collins.<\/p>\n The\u00a0other study<\/a>, led by Tom Misteli, PhD, Director of the Center for Cancer Research, National Cancer Institute, NIH, showed a\u00a090 \u2013 95% reduction of the toxic progerin-producing RNA<\/strong>\u00a0in different tissues after treatment with a drug called LB143. Misteli\u2019s lab found that progerin protein reduction was most effective in the liver, with additional improvements in the heart and aorta.<\/p>\n We now know there are multiple ways to decrease production of the harmful progerin protein using RNA therapeutics. Each study found different stretches of RNA in the mouse models that, when targeted, delivered an effective pathway for treatment, resulting in\u00a0Progeria mice that lived much longer than those treated in previous studies with Zokinvy (lonafarnib)<\/strong>, the only FDA approved drug for children with Progeria. Furthermore, researchers found that a combination treatment with RNA therapeutics and Zokinvy (lonafarnib) reduced progerin protein levels in liver and heart more effectively than either single treatment on its own.<\/p>\n \u201cThese two highly important studies demonstrate the\u00a0major advancements that are now upon us<\/strong>\u00a0in the field of targeted Progeria therapeutics,\u201d said PRF Medical Director, Dr. Leslie Gordon. \u201cI was thrilled to work with these brilliant research groups to advance RNA therapy for children with Progeria. Both are exciting proof-of-principle studies, and\u00a0PRF is excited to forge ahead towards clinical trials<\/strong>\u00a0that apply these treatment strategies.<\/p>\n \u2014<\/p>\n Erdos, M.R., Cabral, W.A., Tavarez, U.L.\u00a0et al.<\/em>\u00a0A targeted antisense therapeutic approach for Hutchinson\u2013Gilford progeria syndrome.\u00a0Nat Med<\/em>\u00a0(2021).\u00a0<\/span>https:\/\/doi.org\/10.1038\/s41591-021-01274-0<\/a><\/p>\n Puttaraju, M., Jackson, M., Klein, S.\u00a0et al.<\/em>\u00a0Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson\u2013Gilford progeria syndrome.\u00a0Nat Med<\/em>\u00a0(2021).\u00a0<\/span>https:\/\/doi.org\/10.1038\/s41591-021-01262-4<\/a><\/p>\n<\/div>\n [\/et_pb_toggle][et_pb_toggle title=”January 2021: Remarkable genetic editing progress in Progeria mouse models” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”January 2021: Remarkable genetic editing progress in Progeria mouse models” _builder_version=”4.24.0″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}”]<\/p>\n The science journal\u00a0Nature<\/em>\u00a0published breakthrough results<\/a>\u00a0demonstrating that genetic editing in a mouse model of Progeria corrected the mutation that causes Progeria in many cells, improved several key disease symptoms and dramatically increased lifespan in the mice.<\/p>\n Co-funded by PRF and co-authored by PRF\u2019s Medical Director Dr. Leslie Gordon, the study found that with a single injection of a base editor programmed to correct the disease-causing mutation, mice survived 2.5 times longer than control untreated Progeria mice, to an age corresponding with the start of old age in healthy mice. Importantly, treated mice also retained healthy vascular tissue\u2014a significant finding, as loss of vascular integrity is a predictor of mortality in children with Progeria.<\/p>\n The study was co-led by world expert in genetic editing, David Liu, PhD, of the Broad Institute, MIT, Jonathan Brown, Assistant Professor of Medicine in the Division of Cardiovascular Medicine at Vanderbilt University, and Francis Collins, MD, PhD, Director of the National Institutes of Health.<\/p>\n \u201cTo see this dramatic response in our Progeria mouse model is one of the most exciting therapeutic developments I have been part of in 40 years as a physician-scientist,\u201d said Dr. Collins.<\/p>\n \u201cFive years ago, we were still finishing the development of the very first base editor,\u201d said Dr. Liu. \u201cIf you had told me then that within five years, a single dose of a base editor could address Progeria in an animal at the DNA, RNA, protein, vascular pathology, and lifespan levels, I would have said \u2018there\u2019s no way.\u2019 It’s a real testament to the dedication of the team that made this work possible.\u201d<\/p>\n Additional preclinical studies are needed to investigate these results, which we hope will one day lead to a clinical trial. Read more about this exciting news in this\u00a0Wall Street Journal<\/em>\u00a0article<\/a>.<\/p>\n<\/div>\n [\/et_pb_toggle][et_pb_toggle title=”November 2020: FDA Approval for lonafarnib (Zokinvy)” open_toggle_background_color=”#f7f7f7″ closed_toggle_text_color=”#ffffff” closed_toggle_background_color=”#00b2e2″ icon_color=”#ffc15e” open_icon_color=”#ffc15e” admin_label=”November 2020: FDA Approval for lonafarnib (Zokinvy)” _builder_version=”4.16″ title_text_color=”#00b2e2″ background_color=”#ffffff” custom_margin=”10px||10px||true” custom_padding=”|50px||50px||true” animation_style=”slide” animation_direction=”top” animation_intensity_slide=”25%” link_option_url_new_window=”on” hover_enabled=”0″ z_index_tablet=”500″ border_width_all=”0px” global_colors_info=”{}” sticky_enabled=”0″]<\/p>\n
<\/strong>A new way to measure progerin, the toxic protein that causes Progeria, has been developed by a team led by PRF co-founder and Medical Director, Dr. Leslie Gordon. With the discovery of this biomarker, which uses blood plasma to measure progerin levels, researchers can understand how treatments are affecting clinical trial participants after a shorter period of time<\/strong>\u00a0and at multiple points along each clinical trial.<\/p>\n
<\/strong>In addition to speeding up future treatment and cure discoveries, this new and innovative way to measure progerin indicates that the long-term benefit of lonafarnib for children with Progeria is greater than previously determined<\/strong>.<\/p>\n
<\/strong>\u2013 Dr. Carolyn Lam, world renowned heart specialist and host of the podcast\u00a0Circulation on the Run,\u00a0<\/em>on the journey that led to these exciting findings.\u00a0<\/em>Hear the full interview<\/strong>\u00a0about the profound impact of this study directly from Dr. Gordon. Listen\u00a0here<\/strong><\/a>\u00a0(<\/a>starting at 6:41).
Hear Dr. Leslie Gordon on Circulation on the Run podcast<\/strong><\/a><\/p>\n![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/03\/Circulation-logo.jpg\"){kind=logo}
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Olsen FJ, Gordon LB, Smoot L, Kleinman ME, Gerhard-Herman M, Hegde SM, Mukundan S, Mahoney T, Massaro J, Ha S, Prakash A. Circulation<\/em>. 2023 Jun 6;147(23):1782-1784. doi: 10.1161\/CIRCULATIONAHA.123.064370. Epub 2023 Jun 5.<\/p>\n
Eriksson M, Haugaa K, Rev\u00eachon G. Circulation<\/em>. 2023 Jun 6;147(23):1745-1747. doi: 10.1161\/CIRCULATIONAHA.123.064765. Epub 2023 Jun 5.<\/p>\n<\/div>\n