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    <title>Cure - Rare Diseases</title>
    <link>https://wewillcure.com/insights/rare-diseases</link>
    <description>Latest content from Cure in Rare Diseases</description>
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      <guid>https://wewillcure.com/insights/rare-diseases/regulatory-and-clinical-strategy/fda-fast-track-personalized-rare-disease-therapies</guid>
      <title><![CDATA[FDA Signals New Fast Track for Personalized Rare-Disease Therapies]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/regulatory-and-clinical-strategy/fda-fast-track-personalized-rare-disease-therapies</link>
      <description><![CDATA[FDA's top leadership just announced a fast-track approval pathway for personalized rare-disease therapies, built around clear biologic mechanisms, early patient improvement, and platform-based evidence from just a few treated cases.]]></description>
      <pubDate>Mon, 17 Nov 2025 05:00:00 GMT</pubDate>
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      <title><![CDATA[AI Is Quietly Solving One of Biotech’s Hardest Problems: Rare Disease Detection]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/ai-and-machine-learning/ai-finding-rare-disease-patients</link>
      <description><![CDATA[A look at AI tools that surface rare-disease patients from limited data—biomarker discovery, EHR scanning, and clinician assistants that aim to shorten the diagnostic delay.]]></description>
      <pubDate>Tue, 28 Oct 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/rare-diseases/entrepreneurship/how-a-teen-turned-his-rare-diagnoses-into-a-rare-movement</guid>
      <title><![CDATA[How a Teen Turned His Rare Diagnoses into a Rare Movement]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/entrepreneurship/how-a-teen-turned-his-rare-diagnoses-into-a-rare-movement</link>
      <description><![CDATA[16-year-old entrepreneur Khartik Uppalapati, co-founder of the RareGen Youth Network, is combining AI innovation and advocacy to reshape how rare diseases are diagnosed and addressed globally.]]></description>
      <pubDate>Tue, 22 Jul 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/cell-and-gene-therapeutics/rare-diseases/federal-gene-therapy-program-expands-access-sickle-cell</guid>
      <title><![CDATA[New Federal Program Expands Access to Gene Therapies for Sickle Cell and More]]></title>
      <link>https://wewillcure.com/insights/cell-and-gene-therapeutics/rare-diseases/federal-gene-therapy-program-expands-access-sickle-cell</link>
      <description><![CDATA[CMS’s new Cell and Gene Therapy Access Model allows states to offer sickle cell gene therapies through federally negotiated contracts, reshaping CGT market for startups and rare disease therapeutics.

]]></description>
      <pubDate>Mon, 21 Jul 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/cell-and-gene-therapeutics/rare-diseases/fda-faster-cell-gene-therapy-reviews</guid>
      <title><![CDATA[FDA Industry Panel Pushes for Faster Cell and Gene Therapy Reviews]]></title>
      <link>https://wewillcure.com/insights/cell-and-gene-therapeutics/rare-diseases/fda-faster-cell-gene-therapy-reviews</link>
      <description><![CDATA[The FDA held a listening session with cell and gene therapy scientists, advocates and entrepreneurs to learn how to improve the regulatory process.]]></description>
      <pubDate>Fri, 13 Jun 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/therapeutics/cell-and-gene-therapeutics/proposed-mrna-bans-alarm-scientists-and-startups</guid>
      <title><![CDATA[Proposed mRNA Bans Alarm Scientists and Startups]]></title>
      <link>https://wewillcure.com/insights/therapeutics/cell-and-gene-therapeutics/proposed-mrna-bans-alarm-scientists-and-startups</link>
      <description><![CDATA[A growing number of state legislatures are proposing laws that would limit and even criminalize mRNA medicines, just as the technology is starting to provide new approaches to diseases ranging from ALS to MD and from pancreatic cancer and HIV/AIDS. ]]></description>
      <pubDate>Wed, 14 May 2025 04:00:00 GMT</pubDate>
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      <category>Article</category>
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      <guid>https://wewillcure.com/insights/rare-diseases/founder-stories/how-a-startup-targets-a-deadly-lung-disease</guid>
      <title><![CDATA[How a Startup Targets a Deadly Lung Disease]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/founder-stories/how-a-startup-targets-a-deadly-lung-disease</link>
      <description><![CDATA[Adiutrix Therapeutics is developing a novel targeted therapy for the lung-stiffening disease known as idiopathic pulmonary fibrosis, offering the potential to be safer and more potent than current treatments.]]></description>
      <pubDate>Fri, 02 May 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/rare-diseases/founder-stories/hibernating-bears-heart-failure-therapy</guid>
      <title><![CDATA[From Hibernating Bears to Humans: A New Therapeutic Path for Heart Failure ]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/founder-stories/hibernating-bears-heart-failure-therapy</link>
      <description><![CDATA[GenKardia's technology is a novel compound targeting Titin — a protein linked to heart stiffness — that takes inspiration from a surprising source: hibernating bears.]]></description>
      <pubDate>Wed, 30 Apr 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/entrepreneurship/founder-stories/mahzi-therapeutics-ceos-advice-for-biotech-female-founders</guid>
      <title><![CDATA[Mahzi Therapeutics CEO's Advice for Biotech Female Founders]]></title>
      <link>https://wewillcure.com/insights/entrepreneurship/founder-stories/mahzi-therapeutics-ceos-advice-for-biotech-female-founders</link>
      <description><![CDATA[Yael Weiss, MD, PhD, founder and CEO of Mahzi Therapeutics, shares her path to biotech leadership, offering insights on navigating the male-dominated industry and championing patient-centered innovation.]]></description>
      <pubDate>Tue, 25 Mar 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/cell-and-gene-therapeutics/rare-diseases/how-beam-therapeutics-success-could-drive-precision-medicine</guid>
      <title><![CDATA[How Beam Therapeutics Success Could Drive Precision Medicine]]></title>
      <link>https://wewillcure.com/insights/cell-and-gene-therapeutics/rare-diseases/how-beam-therapeutics-success-could-drive-precision-medicine</link>
      <description><![CDATA[Beam’s clinical trial is the first to report evidence that a base-editing therapy can rewrite a patient’s genetic code to correct a disease-causing mutation. This milestone now creates a path forward to treat other genetic diseases.]]></description>
      <pubDate>Tue, 18 Mar 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/rare-diseases/how-mahzi-therapeutics-unites-with-patients-for-rare-disease</guid>
      <title><![CDATA[How Mahzi Therapeutics Unites with Patients for Rare Disease]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/how-mahzi-therapeutics-unites-with-patients-for-rare-disease</link>
      <description><![CDATA[Mahzi Therapeutics bridges the gap between patient advocates and researchers, accelerating the development of treatments for rare genetic neurodevelopmental disorders.]]></description>
      <pubDate>Thu, 13 Mar 2025 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/rare-diseases/ai-and-machine-learning/ai-new-gene-variants-poised-to-improve-rare-disease-testing</guid>
      <title><![CDATA[AI, New Gene Variants Poised to Improve Rare Disease Testing]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/ai-and-machine-learning/ai-new-gene-variants-poised-to-improve-rare-disease-testing</link>
      <description><![CDATA[This week's healthcare innovation news focuses on rare diseases: AI speeds diagnoses, variants of 69 genes are newly linked to diseases, GDF15 marker improves mitochondrial disorder testing, and annual FDA-NIH Rare Disease Day postponed.]]></description>
      <pubDate>Fri, 28 Feb 2025 05:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/rare-diseases/founding-and-scaling/rare-disease-startups-face-a-surprising-strategic-dilemma</guid>
      <title><![CDATA[Rare Disease Startups Face a Surprising Strategic Dilemma]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/founding-and-scaling/rare-disease-startups-face-a-surprising-strategic-dilemma</link>
      <description><![CDATA[The Inflation Reduction Act's price negotiation rules are reshaping rare disease drug development strategies, impacting both large and small biotech companies.]]></description>
      <pubDate>Thu, 27 Feb 2025 05:00:00 GMT</pubDate>
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      <category>Article</category>
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      <guid>https://wewillcure.com/insights/rare-diseases/cell-and-gene-therapeutics/duchenne-muscular-dystrophy-treatment-in-transformation</guid>
      <title><![CDATA[Duchenne Muscular Dystrophy Treatment In Transformation]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/cell-and-gene-therapeutics/duchenne-muscular-dystrophy-treatment-in-transformation</link>
      <description><![CDATA[Gene therapy, next-generation steroids, and muscle-protective treatments are among the innovations offering patients with the rare disease Duchenne muscular dystrophy new therapeutic options.]]></description>
      <pubDate>Wed, 26 Feb 2025 05:00:00 GMT</pubDate>
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      <category>Article</category>
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      <guid>https://wewillcure.com/insights/rare-diseases/global-genes-expands-rare-disease-research-and-advocacy</guid>
      <title><![CDATA[Global Genes Expands Rare Disease Research and Advocacy]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/global-genes-expands-rare-disease-research-and-advocacy</link>
      <description><![CDATA[With over 10,000 known rare diseases to address, Global Genes is expanding its RARE-X program to drive innovation.]]></description>
      <pubDate>Wed, 26 Feb 2025 03:00:00 GMT</pubDate>
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      <category>Article</category>
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<item>
      <guid>https://wewillcure.com/insights/rare-diseases/medtech/stephen-kingsmores-quest-to-transform-newborn-screening</guid>
      <title><![CDATA[Stephen Kingsmore’s Quest to Transform Newborn Screening]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/medtech/stephen-kingsmores-quest-to-transform-newborn-screening</link>
      <description><![CDATA[Stephen Kingsmore, MB, ChB, DSc, is enhancing whole genomic sequencing with AI to increase precision and accuracy of newborn screenings.]]></description>
      <pubDate>Mon, 03 Feb 2025 05:00:00 GMT</pubDate>
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      <category>Article</category>
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<item>
      <guid>https://wewillcure.com/insights/rare-diseases/addressing-us-organ-shortage-insights-from-arthur-l-caplan-phd</guid>
      <title><![CDATA[Addressing US Organ Shortage: Insights from Arthur L. Caplan, PhD]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/addressing-us-organ-shortage-insights-from-arthur-l-caplan-phd</link>
      <description><![CDATA[Only a third of patients needing new hearts, lungs, livers, and kidneys get one. Why is there a transplantation gap in the United States, and can new technology and new policies help?]]></description>
      <pubDate>Fri, 10 Jan 2025 05:00:00 GMT</pubDate>
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      <category>Article</category>
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      <guid>https://wewillcure.com/insights/ai-and-machine-learning/rare-diseases/first-ai-designed-drug-improves-ipf-lung-function</guid>
      <title><![CDATA[First AI-Designed Drug Improves IPF Lung Function]]></title>
      <link>https://wewillcure.com/insights/ai-and-machine-learning/rare-diseases/first-ai-designed-drug-improves-ipf-lung-function</link>
      <description><![CDATA[Insilico Medicine reports its investigational SM001-055 - the first drug designed using generative AI - had encouraging topline results in a phase 2A clinical trial.]]></description>
      <pubDate>Tue, 12 Nov 2024 05:00:00 GMT</pubDate>
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      <category>Article</category>
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      <guid>https://wewillcure.com/insights/rare-diseases/cell-and-gene-therapeutics/spotlight-on-2024-biogeneius-challenge-winner-yifan-ding</guid>
      <title><![CDATA[Spotlight on 2024 BioGENEius Challenge Winner: Yifan Ding]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/cell-and-gene-therapeutics/spotlight-on-2024-biogeneius-challenge-winner-yifan-ding</link>
      <description><![CDATA[Developing and validating a novel gene switch, "TREAD," to enhancing CRISPR-based therapies earned high schooler Yifan Ding top honors in the 2024 BioGENEius Challenge.]]></description>
      <pubDate>Fri, 08 Nov 2024 05:00:00 GMT</pubDate>
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<item>
      <guid>https://wewillcure.com/insights/rare-diseases/neurological-health/kif1a-gene-research-advances-rare-neurological-disorder-treatment</guid>
      <title><![CDATA[KIF1A Gene Research Advances Rare Neurological Disorder Treatment]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/neurological-health/kif1a-gene-research-advances-rare-neurological-disorder-treatment</link>
      <description><![CDATA[Arne Gennerich, PhD, Professor of Biochemistry at the Albert Einstein College of Medicine and a 2022 XSeed Award winner, is racing to develop a treatment for patients with rare genetic neurological disorders known as KAND. ]]></description>
      <pubDate>Fri, 18 Oct 2024 04:00:00 GMT</pubDate>
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<item>
      <guid>https://wewillcure.com/insights/therapeutics/rare-diseases/how-evommune-is-redefining-treatments-for-chronic-inflammatory-and</guid>
      <title><![CDATA[How Evommune is Redefining Treatments for Chronic Inflammatory and Autoimmune Diseases]]></title>
      <link>https://wewillcure.com/insights/therapeutics/rare-diseases/how-evommune-is-redefining-treatments-for-chronic-inflammatory-and</link>
      <description><![CDATA[Evommune, led by CEO Luis Peña, is on a mission of tackle one of the world’s leading root cause of deaths: chronic inflammation, which is linked to more than 100 autoimmune diseases.]]></description>
      <pubDate>Wed, 16 Oct 2024 04:00:00 GMT</pubDate>
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<item>
      <guid>https://wewillcure.com/insights/rare-diseases/therapeutics/every-cures-grant-mitchell-on-hopes-and-challenges-for-the-fdas-new-rare</guid>
      <title><![CDATA[Every Cure’s Grant Mitchell on Hopes and Challenges for the FDA’s New Rare Disease Hub]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/therapeutics/every-cures-grant-mitchell-on-hopes-and-challenges-for-the-fdas-new-rare</link>
      <description><![CDATA[The FDA recently announced a new Rare Disease Innovation Hub, designed to expedite development and approval of new treatments for patients with rare diseases. Cure Xchange Challenge winner Every Cure weighs in.]]></description>
      <pubDate>Tue, 08 Oct 2024 04:00:00 GMT</pubDate>
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      <category>Article</category>
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<item>
      <guid>https://wewillcure.com/insights/rare-diseases/founder-stories/insights_article-xseed-award-finalist-dori-thomas-karyat-phd-founder-and-ceo</guid>
      <title><![CDATA[Spotlight on XSeed Award Finalist: Dori Thomas-Karyat, PhD, Founder and CEO of Synthis Therapeutics ]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/founder-stories/insights_article-xseed-award-finalist-dori-thomas-karyat-phd-founder-and-ceo</link>
      <description><![CDATA[Dori Thomas-Karyat, PhD, is developing a potential first-in-class treatment for idiopathic pulmonary fibrosis — a progressive debilitating lung disease with limited therapeutic options.]]></description>
      <pubDate>Wed, 28 Aug 2024 04:00:00 GMT</pubDate>
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      <guid>https://wewillcure.com/insights/cell-and-gene-therapeutics/therapeutics/mvp-insights_article-whitepaper-the-promises-and-perils-of-gene-editing-for</guid>
      <title><![CDATA[The Promises and Perils of Gene Editing for Sickle Cell]]></title>
      <link>https://wewillcure.com/insights/cell-and-gene-therapeutics/therapeutics/mvp-insights_article-whitepaper-the-promises-and-perils-of-gene-editing-for</link>
      <description><![CDATA[]]></description>
      <pubDate>Wed, 05 Jun 2024 04:00:00 GMT</pubDate>
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      <category>White Paper</category>
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      <guid>https://wewillcure.com/insights/rare-diseases/mvp-insights_article-why-rare-diseases-are-really-not-so-rare-and-what-we</guid>
      <title><![CDATA[Why Rare Diseases are Really Not So Rare — and What We Can Learn From Those Living with Them]]></title>
      <link>https://wewillcure.com/insights/rare-diseases/mvp-insights_article-why-rare-diseases-are-really-not-so-rare-and-what-we</link>
      <description><![CDATA[Individuals impacted by rare diseases in myriad ways shared their stories, their insights and how they have turned a diagnosis of a rare disease into an opportunity to advance awareness and the practice of medicine.]]></description>
      <pubDate>Fri, 19 Apr 2024 04:00:00 GMT</pubDate>
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