Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

    ICYMI: With the FDA’s Third Approval for Tissue Agnostic Therapies, Oncologists are Headed to the Next Level in Cancer Treatment
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    ICYMI: With the FDA’s Third Approval for Tissue Agnostic Therapies, Oncologists are Headed to the Next Level in Cancer Treatment

    Since the 1940s oncologists have been focusing on tumors at the location in the body where the tumors originated. According to a recent FDA press release, this is the third…

    Continue Reading ICYMI: With the FDA’s Third Approval for Tissue Agnostic Therapies, Oncologists are Headed to the Next Level in Cancer Treatment
    ICYMI: Study Suggests Female Cancer Patients Have Better Outcomes After Treatment, Experience Worse Side Effects
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    ICYMI: Study Suggests Female Cancer Patients Have Better Outcomes After Treatment, Experience Worse Side Effects

    According to a publication from EurekAlert, a recent study helmed by the Royal Marsden NHS Foundation Trust suggests that female cancer patients tend to live slightly longer following treatment than…

    Continue Reading ICYMI: Study Suggests Female Cancer Patients Have Better Outcomes After Treatment, Experience Worse Side Effects

    The World’s First Editing of DNA in Humans Using CRISPR to Treat Leber Congenital Amaurosis

      Allergan plc, a leading global pharmaceutical company, and its partner, Editas Medicine, Inc. have been given the green light for a clinical trial designed to treat patients with severe…

    Continue Reading The World’s First Editing of DNA in Humans Using CRISPR to Treat Leber Congenital Amaurosis

    Glioblastoma Clinical Trial: The Henry Ford Cancer Institute Enrolls the World’s First Glioblastoma Patient

      According to a recent article in CheckOrphan, glioblastoma (GBM) is considered to be among the deadliest cancers in the world. Currently, treatment options are surgery, chemotherapy, and radiation. In most cases,…

    Continue Reading Glioblastoma Clinical Trial: The Henry Ford Cancer Institute Enrolls the World’s First Glioblastoma Patient

    Improved Guidelines for Patient Advocacy Groups Supporting Rare Diseases and Working with Pharmaceutical Companies

    A study appeared recently in the Orphanet Journal of Rare Diseases reporting that rare diseases affect 350 million people worldwide.  The definition of rare disease differs between the United States…

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    Researchers Might Have Just Made a Breakthrough Discovery for Treating Rare Proteinopathies

    It's common knowledge among researchers who study diseases called toxic proteinopathies that this group of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…

    Continue Reading Researchers Might Have Just Made a Breakthrough Discovery for Treating Rare Proteinopathies

    Attention Biotech and Stem Cell Researchers: Two Potential Grant Programs!

    Patient Worthy wishes to share the following funding opportunities with faculty/ biotech having interest in these areas of research. The two grant programs were announced by the Orphan Disease Center…

    Continue Reading Attention Biotech and Stem Cell Researchers: Two Potential Grant Programs!

    The FDA Will Allow the Most Expensive Therapy in the World to Remain on the Market

    The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug. These…

    Continue Reading The FDA Will Allow the Most Expensive Therapy in the World to Remain on the Market

    Researchers are Working to Repair a Small Step In a Pathway Involved in Neurodegenerative Diseases

    It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…

    Continue Reading Researchers are Working to Repair a Small Step In a Pathway Involved in Neurodegenerative Diseases

    This Time a “First-of-its-Kind” Study is “Out of this World” on Board the Space Station

      As reported recently in PhysOrg by NASA, researchers aboard SpaceX CRS-18 cargo flight are focusing on what triggers Parkinson’s disease and multiple sclerosis, both neurodegenerative diseases. Researchers suspect that…

    Continue Reading This Time a “First-of-its-Kind” Study is “Out of this World” on Board the Space Station

    This Young Man Accurately Diagnosed His Own Disorder But Doctors Did Not Believe His Diagnosis

    Doug Lindsay ran track in high school. According to an article published by CNN Health, Doug had big plans as a senior at Rockhurst University in Kansas City where he…

    Continue Reading This Young Man Accurately Diagnosed His Own Disorder But Doctors Did Not Believe His Diagnosis

    A Brief Discussion of How Cell and Gene Therapies are Being Improved for Future Generations

      While the last few years have seen major advances in all areas of the pharmaceutical industry, some of the newest and brightest talents are now focusing on cell and…

    Continue Reading A Brief Discussion of How Cell and Gene Therapies are Being Improved for Future Generations

    DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases

    Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…

    Continue Reading DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases

    Research Team Identifies Diagnostic Errors in Three Classes of Disease Associated with a High Death Rate or Permanent Disability

      The number of people in the U.S. who are permanently disabled or who die each year as a result of misdiagnosis is estimated to be over 100,000. EurekAlert recently reported…

    Continue Reading Research Team Identifies Diagnostic Errors in Three Classes of Disease Associated with a High Death Rate or Permanent Disability

    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,…

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

    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…

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

    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…

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