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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A Tale of Two Sisters With Cystic Fibrosis Highlights the Barriers to Treatment Access That Still Remain
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A Tale of Two Sisters With Cystic Fibrosis Highlights the Barriers to Treatment Access That Still Remain

According to a story from the Sunday Post, sisters Shona and Kirsty Young, aged 24 and 29 years respectively, were both born with the devastating rare disease cystic fibrosis. However,…

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Zogenix Acquires Modis in $400 Million Buyout, Adds Experimental TK2 mDNA Depletion Syndrome Treatment to Pipeline

According to a press release from California-based biotechnology company Zogenix, the Company recently completed a multi-million dollar acquisition of Modis Therapeutics. Before its acquisition, Modis focused on the development of…

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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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FDA Begins Priority Review of Xtandi, a Drug Candidate for Metastatic Hormone-Sensitive Prostate Cancer

According to a press release from American pharmaceutical giant Pfizer, Inc. the FDA recently agreed to review a supplemental New Drug Application for Xtandi. Xtandi was first approved for the…

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FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy
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FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy

According to a press release from American biotechnology company Fortress Biotech, the American Food and Drug Administration (FDA) recently awarded Regenerative Medicine Advanced Therapy designation to the experimental gene therapy…

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Beta Blocker Fails to Stop Hereditary Hemorrhagic Telangiectasia-Associated Nosebleeds
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Beta Blocker Fails to Stop Hereditary Hemorrhagic Telangiectasia-Associated Nosebleeds

According to a recent study published in Nature Research, a team of scientists studied the efficacy of timolol in treating nosebleeds associated with hereditary hemorrhagic telangiectasia. Timolol is a beta…

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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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CPRIT Awards Emtora Biosciences $3 Million Grant to Develop Investigational Familial Adenomatous Polyposis Drug
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CPRIT Awards Emtora Biosciences $3 Million Grant to Develop Investigational Familial Adenomatous Polyposis Drug

According to a recent press release, Texas-based biotechnology company Emtora Biosciences was chosen earlier this month to receive a $3 million grant from the Cancer Prevention and Research Institute of…

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Good Morning America Spotlights Girl with Rare Disease Mesmerized by Inclusive Beauty Ad

Recently, Good Morning America demonstrated the importance of showing children with disabilties examples of diversity in the media. Carolyn Anderson shared with the popular morning show how her 4-year-old-daughter Maren…

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