{"id":33696,"date":"2020-08-14T14:53:56","date_gmt":"2020-08-14T20:53:56","guid":{"rendered":"https:\/\/www.uchealth.org\/today\/?p=33696"},"modified":"2020-08-24T09:54:56","modified_gmt":"2020-08-24T15:54:56","slug":"givlaari-prescription-drug-provides-newfound-freedom-for-acute-intermittent-porphyria-patient","status":"publish","type":"post","link":"https:\/\/www.uchealth.org\/today\/givlaari-prescription-drug-provides-newfound-freedom-for-acute-intermittent-porphyria-patient\/","title":{"rendered":"Givlaari, a new prescription drug, changed her life"},"content":{"rendered":"<div style=\"margin-top: 0px; margin-bottom: 0px;\" class=\"sharethis-inline-share-buttons\" ><\/div><p>Hannah Moraski has always been independent, courageous and someone who exemplifies the word tattooed across her forearm: Fighter.<\/p>\n<p>She has vast appreciation for her newfound freedom (even in a pandemic), a new prescription drug, Givlaari, that has changed her life and Justin Smith, a handsome young man whom she married on Aug. 21.<\/p>\n<p>On a recent camping trip with Justin, one of the first she has been able to take in years, Moraski, 26, spent several minutes taking in the glory that is <a href=\"https:\/\/en.wikipedia.org\/wiki\/Collegiate_Peaks\" target=\"_blank\" rel=\"noopener noreferrer\">Colorado\u2019s Collegiate Peaks <\/a>near Buena Vista.<\/p>\n<figure id=\"attachment_33697\" aria-describedby=\"caption-attachment-33697\" style=\"width: 640px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-33697 size-large\" src=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14103932\/hannahmoraski2.jpgtiny.webp\" alt=\"Hannah Moraski and her soon-to-be husband, Justin Smith, practice archery. Because of a new prescription drug, Givlaari, Hannah has a vast appreciation for her newfound freedom (even in a pandemic).\" width=\"640\" height=\"438\" srcset=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14103932\/hannahmoraski2.jpgtiny.webp 1200w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14103932\/hannahmoraski2.jpgtiny-300x205.webp 300w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14103932\/hannahmoraski2.jpgtiny-1024x701.webp 1024w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14103932\/hannahmoraski2.jpgtiny-768x525.webp 768w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14103932\/hannahmoraski2.jpgtiny-150x103.webp 150w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14103932\/hannahmoraski2.jpgtiny-200x137.webp 200w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><figcaption id=\"caption-attachment-33697\" class=\"wp-caption-text\">Hannah Moraski and her fianc\u00e9, Justin Smith, practice archery. Because of a new prescription drug, Givlaari, Hannah has a vast appreciation for her newfound freedom (even in a pandemic). Photos:\u00a0courtesy Hannah Moraski.<\/figcaption><\/figure>\n<p>\u201cJust looking out over the mountains and knowing that you are alive and being grateful,\u2019\u2019 she said. \u201c\u2026 I am so happy to have my life back.\u2019\u2019<\/p>\n<p>Five years ago, Moraski became suddenly ill. Bedridden, she couldn\u2019t eat, shower or walk. Over a few months, she made frequent trips to the Emergency Department and took painkillers for debilitating pain in her back, legs and abdomen. She was miserable.<\/p>\n<p>Eventually, Moraski was diagnosed with a rare blood disease \u2013 <a href=\"https:\/\/rarediseases.info.nih.gov\/diseases\/5732\/acute-intermittent-porphyria\" target=\"_blank\" rel=\"noopener noreferrer\">Acute Intermittent Porphyria<\/a> \u2013 a genetic disease that affects only five in every 100,000 people.<\/p>\n<p>\u201cYou\u2019d be hard-pressed to find any of us in the community who have severe acute intermittent porphyria patients,\u201d said Dr. Uchenna Njiaju, a hematology and oncology specialist who cares for Moraski at <a href=\"https:\/\/www.uchealth.org\/locations\/uchealth-memorial-hospital-central\/\">UCHealth Memorial Hospital Central<\/a>. \u201cSo, there\u2019s nobody, probably, except for expert geneticists, a couple around the country, who can lay claim to having treated a hundred over two years or that sort of thing. We all have a sprinkling, because it\u2019s so rare.\u201d<\/p>\n<p>In most people, symptoms never manifest and people live their lives not knowing they have acute intermittent porphyria. But, Njiaju says, Hannah had a rather aggressive presentation and course with AIP. For Hannah, the attacks sent her to the hospital for week-long stays. During one stay, she suffered cardiac arrest.<\/p>\n<p>For the past five years, Moraski has been shackled to twice-a-week visits to UCHealth Memorial Hospital, where she received infusions of panhematin, a drug that helps replace what her blood can\u2019t produce. Each session lasted 2 to 3 hours, and she passed the time reading, knitting and talking to her nurse.<\/p>\n<p>Moraski could never travel far from the hospital. She got used to thinking she\u2019d never be able to take a vacation, hold a full-time job or even go camping for more than a few days at a time. The need to be close to a hospital to receive the hours-long infusions was her first priority.<\/p>\n<p>In January, a drug called <a href=\"https:\/\/porphyrianews.com\/2020\/06\/12\/givlaari-markedly-reduces-aip-attacks-phase-3-trial-shows\/\" target=\"_blank\" rel=\"noopener noreferrer\">Givlaari<\/a> was released. Now, instead of spending at least 6 hours a week sitting in a chair to receive infusions, Moraski gets a shot once a month. It takes about 30 minutes.<\/p>\n<p>\u201cIt&#8217;s just a little shot and I don\u2019t have to sit here,\u2019\u2019 she said. \u201cWith everything going on in the world, it really couldn\u2019t have happened at a better time.<\/p>\n<figure id=\"attachment_33698\" aria-describedby=\"caption-attachment-33698\" style=\"width: 640px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-33698 size-large\" src=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14104114\/hannahmoraski.jpgtiny.webp\" alt=\"Hannah Moraski and Justin Smith. In January, a drug called Givlaari was released, which has changed Hannah's life.\" width=\"640\" height=\"640\" srcset=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14104114\/hannahmoraski.jpgtiny.webp 1000w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14104114\/hannahmoraski.jpgtiny-300x300.webp 300w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14104114\/hannahmoraski.jpgtiny-150x150.webp 150w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14104114\/hannahmoraski.jpgtiny-768x768.webp 768w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14104114\/hannahmoraski.jpgtiny-200x200.webp 200w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><figcaption id=\"caption-attachment-33698\" class=\"wp-caption-text\">Hannah Moraski and Justin Smith. In January, a drug called Givlaari was released, which has changed Hannah&#8217;s life.<\/figcaption><\/figure>\n<p>\u201cIt didn\u2019t occur to me until I got this drug what I was really missing in life and what I now have back,\u2019\u2019 she said. \u201cIt\u2019s really hard to take anything for granted and little things like camping, just looking out over the mountains and knowing that you are alive and being grateful that this drug exists.\u2019\u2019<\/p>\n<p>Moraski is working again. Previously, she could work only about one day a month. She dreams of returning to college and pursuing an education in psychology\/mental health.<\/p>\n<p>\u201cI really want to help people in that way, and I think my own personal struggle has made me realize that I want to help people that much more. It\u2019s more than just helping, it\u2019s connecting and understanding and being a voice for people who do not feel as though they can be a voice for themselves,\u2019\u2019 she said.<\/p>\n<p>For her doctor, watching Hannah&#8217;s progress has been a joy.<\/p>\n<p>&#8220;Hannah has really come a long way. Her strength and resilience through challenges that would demoralize most other people, are truly inspiring. Hannah always has a lovely smile on her face. It&#8217;s been such an honor to partner with her on this journey. I&#8217;m just so happy to see her healthy, having less frequent infusion appointments and more time away from the hospital to live her life,&#8221; Njiaju said.<\/p>\n<p>In recent weeks, the biggest thing in her life has been her wedding on\u00a0Aug. 21 to Justin, who has stuck by her from the day she was diagnosed.<\/p>\n<p>\u201cJustin and I had always had to plan things around my infusions, everything was controlled around infusions. That\u2019s the biggest thing, I\u2019m so grateful for his support. It\u2019s changed his life too,\u2019\u2019 she said.<\/p>\n<p>\u201cHe has always stuck by my side and now he can enjoy things in his life too. I am so lucky to have him, and I can give back to him and I feel like I am actually going to be the wife he deserves. He has done so much for me over the years and now I can give back, I can finally do it thanks to the drug.\u2019\u2019<\/p>\n<p>The two traveled to Vermont, where Moraski was born and raised, for a small wedding among family and a few friends. It\u2019s the first time that Moraski has been back to Vermont since she moved to Colorado five years ago. Because of the coronavirus pandemic, the couple will opt for a smaller crowd and delay their honey moon until the environment for travel is safer.<\/p>\n<div class=\"su-callout-box col-xs-6 col-sm-6 right\" style=\"background-color:#dce4e7; color:#2e3b44;\">\n<p><strong>Acute intermittent porphyria\u00a0<\/strong><\/p>\n<p><span style=\"font-family: 'Arial',sans-serif\">Dr. Uchenna Njiaju says the main problem in AIP is that there is deficiency of an enzyme called porphobilinogen deaminase. \u00a0This enzyme helps to break down molecules called porphyrins that form in the process of manufacturing heme, the main pigment that enable red cells to carry oxygen.<\/span><\/p>\n<p><span style=\"font-family: 'Arial',sans-serif\">When porphyrins accumulate excessively in AIP, they cause attacks and problems in the nervous tissues. \u00a0The main way the genetic code instructs the body to make proteins is through molecules we called mRNA.<\/span><\/p>\n<p><span style=\"font-family: 'Arial',sans-serif\">Givlaari is a small molecule, that interferes with the genetic message (mRNA) leading to manufacture of nerve system poisons in AIP. <\/span><\/p>\n<p><span style=\"font-family: 'Arial',sans-serif\">&#8220;By using Givlaari, we have fewer of these toxic molecules floating around and so we can prevent AIP attacks,&#8221; Njiaju says. \u00a0\u00a0<\/span><\/p>\n<\/div>\n<p>\u201cPossibly next year, we want to plan a big honeymoon. Possibly next year we will be going and doing a cruise, which is something that I\u2019ve never done. We never thought it would be possible to travel, to go on an actual cruise.\u2019\u2019<\/p>\n<p>\u201cThis new drug opens up a whole new world. It\u2019s literally living life. It\u2019s like I had these couple of years of struggling and it has paid off,\u2019\u2019 she said.<\/p>\n<p>At first, Moraski admits, she was nervous to take Givlaari because she was not sure her body would accept the drug. Some people have side effects \u2013 high liver enzymes or certain rashes &#8211; around month 3 or 4.<\/p>\n<figure id=\"attachment_19419\" aria-describedby=\"caption-attachment-19419\" style=\"width: 191px\" class=\"wp-caption alignleft\"><img loading=\"lazy\" decoding=\"async\" class=\"size-full wp-image-19419\" src=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2018\/11\/08135206\/Njiaju.webp\" alt=\"\" width=\"191\" height=\"244\" srcset=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2018\/11\/08135206\/Njiaju.webp 191w, https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2018\/11\/08135206\/Njiaju-117x150.webp 117w\" sizes=\"auto, (max-width: 191px) 100vw, 191px\" \/><figcaption id=\"caption-attachment-19419\" class=\"wp-caption-text\">Dr. Uchenna Njiaju<\/figcaption><\/figure>\n<p>\u201cI want everyone else to be able to get the medical care they need and experience life, whether it is with AIP or any other disease. Often, disease goes unnoticed and people just suffer in silence and I don\u2019t want those people to suffer in silence,\u2019\u2019 Moraski said.<\/p>\n<p>Moraski has been influential in social media circles among people who have AIP. When the newly diagnosed are looking for a physician, she often recommends Dr. Njiaju.<\/p>\n<p>\u201cDr. Njiaju has really made a name for herself. She\u2019s become an expert on AIP. She\u2019ll call me personally and she takes phone calls from my mom. The two of them are in constant communication and I don\u2019t think I would be able to go through this without Dr. Njiaju.\u2019\u2019<\/p>\n<p>Moraski has always appreciated Njiaju\u2019s direct but compassionate approach.<\/p>\n<p>\u201cShe\u2019s always kind of told me straight up. She\u2019s been honest and straight-forward but also very loving. And that\u2019s what you need in a doctor. She knows what she is doing and she is incredibly intelligent. She\u2019s an amazing person.\u2019\u2019<\/p>\n<p>After regular visits to UCHealth Memorial Hospital two times a week for five years, Moraski is happy to be able to take time for herself, work and go camping with Justin.<\/p>\n<p>\u201cI could not have asked for better nurses. The people here, they have seen my journey and they\u2019ve been through the progress with me,\u2019\u2019 Moraski said. \u201cThey are so happy for me. I love them to death, but I am so happy that I don\u2019t have to see them.\u2019\u2019<\/p>\n<p>Moraski was radiant as she walked down the aisle. She and Justin exchanged rings and recited their vows, After their wedding in Vermont, they began their journey together, free to go anywhere their hearts desire.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Hannah Moraski has always been independent, courageous and someone who exemplifies the word tattooed across her forearm: Fighter. She has vast appreciation for her newfound freedom (even in a pandemic), a new prescription drug, Givlaari, that has changed her life and Justin Smith, a handsome young man whom she married on Aug. 21. On a [&hellip;]<\/p>\n","protected":false},"author":20,"featured_media":33697,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_relevanssi_hide_post":"","_relevanssi_hide_content":"","_relevanssi_pin_for_all":"","_relevanssi_pin_keywords":"","_relevanssi_unpin_keywords":"","_relevanssi_related_keywords":"","_relevanssi_related_include_ids":"","_relevanssi_related_exclude_ids":"","_relevanssi_related_no_append":"","_relevanssi_related_not_related":"","_relevanssi_related_posts":"","_relevanssi_noindex_reason":"","footnotes":""},"categories":[5],"tags":[1024,63],"class_list":["post-33696","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-innovative-care","tag-blood-disorders-disease-care-hematology","tag-pharmacy"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.7 (Yoast SEO v27.7) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>Givlaari, new prescription drug, changed her life - UCHealth Today<\/title>\n<meta name=\"description\" content=\"Hannah Moraski has vast appreciation for her newfound freedom (even in a pandemic), thanks to a new prescription drug, Givlaari, that has changed her life.\" \/>\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.uchealth.org\/today\/givlaari-prescription-drug-provides-newfound-freedom-for-acute-intermittent-porphyria-patient\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Givlaari, a new prescription drug, changed her life\" \/>\n<meta property=\"og:description\" content=\"Hannah Moraski has vast appreciation for her newfound freedom (even in a pandemic), thanks to a new prescription drug, Givlaari, that has changed her life.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.uchealth.org\/today\/givlaari-prescription-drug-provides-newfound-freedom-for-acute-intermittent-porphyria-patient\/\" \/>\n<meta property=\"og:site_name\" content=\"UCHealth Today\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/uchealthorg\/\" \/>\n<meta property=\"article:published_time\" content=\"2020-08-14T20:53:56+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2020-08-24T15:54:56+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/uchealth-wp-uploads.s3.amazonaws.com\/wp-content\/uploads\/sites\/6\/2020\/08\/14103932\/hannahmoraski2.jpgtiny.jpg\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Erin Emery, UCHealth\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@uchealth\" \/>\n<meta name=\"twitter:site\" content=\"@uchealth\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Erin Emery, UCHealth\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"8 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/www.uchealth.org\\\/today\\\/givlaari-prescription-drug-provides-newfound-freedom-for-acute-intermittent-porphyria-patient\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.uchealth.org\\\/today\\\/givlaari-prescription-drug-provides-newfound-freedom-for-acute-intermittent-porphyria-patient\\\/\"},\"author\":{\"name\":\"Erin Emery, UCHealth\",\"@id\":\"https:\\\/\\\/www.uchealth.org\\\/today\\\/#\\\/schema\\\/person\\\/f989ee92cb6da7cfb7f697db1c6771b3\"},\"headline\":\"Givlaari, a new prescription drug, changed her life\",\"datePublished\":\"2020-08-14T20:53:56+00:00\",\"dateModified\":\"2020-08-24T15:54:56+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.uchealth.org\\\/today\\\/givlaari-prescription-drug-provides-newfound-freedom-for-acute-intermittent-porphyria-patient\\\/\"},\"wordCount\":1557,\"publisher\":{\"@id\":\"https:\\\/\\\/www.uchealth.org\\\/today\\\/#organization\"},\"image\":{\"@id\":\"https:\\\/\\\/www.uchealth.org\\\/today\\\/givlaari-prescription-drug-provides-newfound-freedom-for-acute-intermittent-porphyria-patient\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/uchealth-wp-uploads.s3.amazonaws.com\\\/wp-content\\\/uploads\\\/sites\\\/6\\\/2020\\\/08\\\/14103932\\\/hannahmoraski2.jpgtiny.webp\",\"keywords\":[\"Blood disorders &amp; 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