{"id":10189,"date":"2019-08-07T07:54:30","date_gmt":"2019-08-07T02:24:30","guid":{"rendered":"https:\/\/www.rootsanalysis.com\/blog\/?p=10189"},"modified":"2026-07-30T18:54:59","modified_gmt":"2026-07-30T13:24:59","slug":"mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases","status":"publish","type":"post","link":"https:\/\/www.rootsanalysis.com\/blog\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\/","title":{"rendered":"Mitochondrial Disease Therapies: Unlocking Treatment for Orphan Diseases"},"content":{"rendered":"<div class=\"wp-block-image\">\n<figure class=\"alignleft\"><img loading=\"lazy\" decoding=\"async\" width=\"150\" height=\"150\" src=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2019\/08\/Mitochondrial-Diseases-150x150.jpg\" alt=\"\" class=\"wp-image-10191\"\/><\/figure>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\">In the US, <a href=\"https:\/\/www.rootsanalysis.com\/reports\/view_document\/mitochondrial-disease-therapies-market-2019-2030\/264.html\">mitochondrial diseases<\/a> are known to annually affect 1 in 5,000 individuals. It is also estimated that 1,000-4,000 children are born with a mitochondrial disease every year, in the same region. Primary mitochondrial diseases are usually difficult to diagnose. Such clinical conditions are rare and are known to affect multiple parts of the body, including neurons and nerves, kidneys, heart, liver, eyes, ears, and pancreas. It is worth highlighting that over 300 mutations that are associated with various mitochondrial diseases have been identified.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">I have highlighted below some of the most interesting insights from our study. You can also access additional details here.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Despite various obstacles, mostly related to accurate diagnosis, currently, a number of personalized and off the shelf therapeutic options are being investigated for the treatment of primary mitochondrial diseases <\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Presently, around 60 therapies, including certain repositioned \/ repurposed drugs, are being evaluated for the treatment of primary mitochondrial diseases by both industry players and academic institutions, worldwide<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Majority of drug \/ therapy candidates are in the early to mid stages of clinical development, and are designed to treat a variety of mitochondrial diseases exploiting the oral route of administration<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Efforts to innovate in this field is evident from the R&amp;D activity, which can be quantified in terms of volume of published scientific literature, focused on multiple diseases and having diverse aims and objectives <\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>A number of prominent scientists from renowned universities, owing to their involvement in clinical development efforts, have emerged as key opinion leaders<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Over the time, several non-profit organizations have extended financial support for research efforts in this domain; the current focus is on understanding the disease etiopathology and developing effective therapies<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>The success of the first approved therapy (Raxone\u00ae) will determine the current state of the market; the future growth is likely to be driven by the safety and efficacy outcomes of the late-stage molecules, once approved<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>The estimated future opportunity is likely to be distributed across diverse types of therapies, different classes of therapeutic interventions and routes of administration<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\">To learn more, please click here. For any queries \/ suggestions, please do not hesitate to contact us at <a href=\"mailto:sales@rootsanalysis.com\">sales@rootsanalysis.com<\/a>. Alternatively, you can place an Order by filling this <a href=\"https:\/\/www.rootsanalysis.com\/uploads\/documents\/Mitochondrial%20Disease%20Therapies%20Market,%202019-2030%20(Order%20Form).pdf\">Order Form<\/a>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n","protected":false},"excerpt":{"rendered":"<p>In the US, mitochondrial diseases are known to annually affect 1 in 5,000 individuals. It is also estimated that 1,000-4,000 children are born with a mitochondrial disease every year, in the same region. Primary mitochondrial diseases are usually difficult to diagnose. Such clinical conditions are rare and are known to affect multiple parts of the [&hellip;]<\/p>\n","protected":false},"author":8,"featured_media":18914,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[344],"tags":[345],"class_list":["post-10189","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-mitochondrial-disease","tag-mitochondrial-disease"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.3 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Mitochondrial Disease Therapies: Unlocking Treatment... | Blog<\/title>\n<meta name=\"description\" content=\"In the US, mitochondrial diseases are known to annually affect 1 in 5,000 individuals. 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It is also estimated that 1,000-4,000...\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.rootsanalysis.com\/blog\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\/\" \/>\n<meta property=\"og:site_name\" content=\"Blog\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/RootsAnalysis\/\" \/>\n<meta property=\"article:published_time\" content=\"2019-08-07T02:24:30+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2026-07-30T13:24:59+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.rootsanalysis.com\/blog\/wp-content\/uploads\/2019\/08\/Roots-Analysis-FeaturedImage-Mitochondrial-Diseases.webp\" \/>\n\t<meta property=\"og:image:width\" content=\"150\" \/>\n\t<meta property=\"og:image:height\" content=\"150\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/webp\" \/>\n<meta name=\"author\" content=\"Arun Gupta\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@RootsAnalysis\" \/>\n<meta name=\"twitter:site\" content=\"@RootsAnalysis\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Arun Gupta\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"3 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\\\/\"},\"author\":{\"name\":\"Arun Gupta\",\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/#\\\/schema\\\/person\\\/af9198bf467c6e126793d9da458a1f62\"},\"headline\":\"Mitochondrial Disease Therapies: Unlocking Treatment for Orphan Diseases\",\"datePublished\":\"2019-08-07T02:24:30+00:00\",\"dateModified\":\"2026-07-30T13:24:59+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\\\/\"},\"wordCount\":374,\"publisher\":{\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/#organization\"},\"image\":{\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/wp-content\\\/uploads\\\/2019\\\/08\\\/Roots-Analysis-FeaturedImage-Mitochondrial-Diseases.webp\",\"keywords\":[\"Mitochondrial Disease\"],\"articleSection\":[\"Mitochondrial Disease\"],\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\\\/\",\"url\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\\\/\",\"name\":\"Mitochondrial Disease Therapies: Unlocking Treatment... | Blog\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\\\/#primaryimage\"},\"image\":{\"@id\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/mitochondrial-disease-therapies-unlocking-treatment-for-orphan-diseases\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/www.rootsanalysis.com\\\/blog\\\/wp-content\\\/uploads\\\/2019\\\/08\\\/Roots-Analysis-FeaturedImage-Mitochondrial-Diseases.webp\",\"datePublished\":\"2019-08-07T02:24:30+00:00\",\"dateModified\":\"2026-07-30T13:24:59+00:00\",\"description\":\"In the US, mitochondrial diseases are known to annually affect 1 in 5,000 individuals. 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