At Age 6, A Boy Finally Discovers His Disease: He’s The First To Be Diagnosed With It
PNH causes the immune system to destroy red blood cells. Source: pixabay.com

At Age 6, A Boy Finally Discovers His Disease: He’s The First To Be Diagnosed With It

Originally reported in People Magazine, a family struggled for years to figure out what was happening to their son, Cohen Bramlee, who for years received his nutrients from a central…

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UPDATE: Trump Admin Reconsiders Decision to Deport Migrants Receiving Life-Saving Treatment 

Last week, we spotlighted a New York Times story about how under the Trump Administration’s new immigration policy, six migrants undergoing lifesaving treatments would be abruptly deported; including a young woman…

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Patient Advocacy Groups Call for Indian Health Ministry to Implement Interim Care Policy for Rare Disease Patients
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Patient Advocacy Groups Call for Indian Health Ministry to Implement Interim Care Policy for Rare Disease Patients

According to a recent publication from Moneycontrol, patient advocacy groups in India are calling for the country's Minister of Health and Family Welfare, Harsh Vardhan, to establish a provisional system…

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Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome
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Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome

According to a publication from the Korea Times, the Eone Diagnomics Genome Center in Incheon, South Korea, is working to improve patients' accessibility to their own genetic sequence. Researchers believe…

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A Recent Discovery Could Make Cancer Treatments More Effective
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A Recent Discovery Could Make Cancer Treatments More Effective

According to a story from news-medical.net, scientists may have made a significant breakthrough discovery that could make cancer treatment more effective. The discovery involves a new method that breaks through…

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ICYMI: An Experimental Treatment for POMC and LEPR Deficiency Obesity Appears Effective

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., has recently announced the results from two phase 3 clinical trials. These trials were testing the company's experimental…

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PointR Data Merges into Mateon Therapeutics — Company Will Focus on Using Data Analysis and AI to Develop New Treatments
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PointR Data Merges into Mateon Therapeutics — Company Will Focus on Using Data Analysis and AI to Develop New Treatments

According to a press release from Mateon Therapeutics, Mateon's recent merger agreement with PointR Data establishes plans to combine the use of artificial intelligence and data collection for the development…

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Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation
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Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company CrystalGenomics, Inc. recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational drug candidate CG-745 Orphan…

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Rapid Whole Genome Sequencing is Getting Patients With Rare Genetic Disorders Diagnosed More Quickly
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Rapid Whole Genome Sequencing is Getting Patients With Rare Genetic Disorders Diagnosed More Quickly

According to a story from the National Center for Advancing Translational Sciences (NCATS), research supported by the center is making it possible for kids born with serious, rare genetic disorders…

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Watch: Netflix Docuseries ‘Diagnosis’ About the Struggle of Diagnosing Mysterious Diseases 

Netflix debuted a documentary series this month called 'Diagnosis' -- based on a New York Times Magazine column -- which follows individuals with or caregiving for patients with a mysterious,…

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FDA Grants Breakthrough Therapy Designation to Investigational Hepatitis D Treatment
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FDA Grants Breakthrough Therapy Designation to Investigational Hepatitis D Treatment

According to a press release from Eiger Biopharmaceuticals, the American Food and Drug Administration (FDA) recently granted the Company's experimental hepatitis delta virus drug (hepatitis D), peginterferon lambda, breakthrough therapy…

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Synlogic Halts Development of Investigational Hyperammonemia Treatment SYNB1020
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Synlogic Halts Development of Investigational Hyperammonemia Treatment SYNB1020

According to a press release from Synlogic Therapeutics, the Company recently halted development of its investigational hyperammonemia treatment SYNB1020 after an unsuccessful phase 1/2 clinical study. About Hyperammonemia Hyperammonemia is…

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