Watch: New York Giants Running Back Grants Wish for Teen Living with Ewing’s Sarcoma

15-year-old George Taylor reached out to NY Giants Running Back Saquon Barkley, telling the star NFL player how he's an inspiration to him for when he undergoes his chemotherapy for Ewing's sarcoma,…

Continue Reading Watch: New York Giants Running Back Grants Wish for Teen Living with Ewing’s Sarcoma
Rare Disease Patients in Uttar Pradesh Forced to Abandon Treatment Amid Funding Crisis
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Rare Disease Patients in Uttar Pradesh Forced to Abandon Treatment Amid Funding Crisis

According to a publication from ET Healthworld, some 21 rare disease patients in India's northern state of Uttar Pradesh are still awaiting governmental financial assistance to which they are legally…

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FDA Clears Phase 2 Trial for Possible Idiopathic Pulmonary Fibrosis Drug

According to a story from BioPortfolio, the biopharmaceutical company Genkyotex has recently announced that the US Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application.…

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Since 1982, Over 16 Million People Have Been Cured of Hansen’s Disease Worldwide, But a Major Struggle Remains in Brazil

  According to a report recently published in the Inter Press Service, since the advent of multidrug therapy in 1982, over sixteen million people have been cured of Hansen’s disease,…

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Experimental Netherton Syndrome Treatment Receives Rare Pediatric Disease Designation from FDA
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Experimental Netherton Syndrome Treatment Receives Rare Pediatric Disease Designation from FDA

According to a press release from LifeMax Laboratories, Inc., the Food and Drug Administration (FDA) has granted the Company's experimental Netherton syndrome drug LM-030 (licensed from Novartis) Rare Pediatric Disease…

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CRISPR Genome Editing and Stem Cell Technology Uncovered the Cause of the Baby’s Heart Disorder

  Tatiana Legkiy was only two months old when she was rushed to a hospital in San Francisco after an echocardiogram showed that her heart was malfunctioning. A recent article in…

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Doctors Are Unable to Diagnose Many Rare Diseases. Genome Sequencing is Changing That Scenario

After twenty-two years physician Huang Chun-jung finally learned why his vision and hearing have fallen to thirty percent of normalcy, thanks to research conducted by the National Health Research Institutes…

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Alberta Diabetes Institute to Study Use of Anti-Obesity Drug in Alström and Bardet-Biedl Patients
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Alberta Diabetes Institute to Study Use of Anti-Obesity Drug in Alström and Bardet-Biedl Patients

According to a publication from EurekAlert, a clinical trial of setmelanotide, an experimental anti-obesity drug, is set to begin soon at the Alberta Diabetes Institute (of the University of Alberta).…

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Possible Treatment for Homozygous Familial Hypercholesterolemia Earns Orphan Drug Designation
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Possible Treatment for Homozygous Familial Hypercholesterolemia Earns Orphan Drug Designation

According to a story from bloomberg.com, the drug development company Arrowhead Pharmaceuticals, Inc. recently announced that its experimental product candidate AR0-ANG3 has earned Orphan Drug designation from the US Food…

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Antibodies: The Key to Fighting Sickle Cell Anemia? Approval Could be Just Months Away
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Antibodies: The Key to Fighting Sickle Cell Anemia? Approval Could be Just Months Away

According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This…

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Huntsman Lab Research Team Discovers that Selinexor May Benefit Myelofibrosis Patients Who have No Other Curative Options

  Myelofibrosis (MF) comes under the heading of rare cancer. A recent article in Newswise, University of Utah, describes the disease as the failure of bone marrow to produce normal…

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