Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

    FDA Approval Was Granted For a Duchenne Muscular Dystrophy Drug Amid Internal Disagreements

      The accelerated approval granted by the FDA for the Duchenne muscular dystrophy drug eteplirsen, developed by Sarepta Therapeutics, was granted only after internal disagreements and protests had been resolved.…

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    FDA Approves a Combination of Drugs for Diffuse Large B-cell Lymphoma

      Diffuse large B-cell lymphoma  (DLBCL) is a common type of non-Hodgkins lymphoma. World Pharma News recently reported that the FDA approved the first chemoimmunotherapy, Polivy, for DLBCL in combination with…

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    The Sudden Closure of Northwestern University’s Stem Cell Therapy Clinic Raises Questions That Remain Unanswered

    According to a recent article in The Scientist, Northwestern Medicine’s successful autoimmune and immunotherapy program is winding down and will be closed by the end of the year. The program,…

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    Patients in Mayo Clinic’s RIGHT Study Carried One or More Genetic Variants That May Affect Their Response to Medication

      Pharmacogenomics plays an important role in predicting whether patients will benefit from certain medications or if the medication will cause serious side effects. The process involves the way in…

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    Biohacking: A Small Group of Amateur Biologists Are Hoping to Market Their Knockoff of a One Million Dollar Gene Therapy

      According to a recent article in Archive Today, an international band of amateur biologists, or "biohackers" announced that they intend to eventually market a knock-off of Glybera, a one…

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    CRISPR Gene Editing Promises to Cure 10,000 diseases. Now in Its First Human Clinical Trial. Will it Eventually Deliver?

      Clustered Regularly Interspaced Short Palindromic Repeats is more commonly known as CRISPR. As reported recently in Science News, the CRISPR/Cas9 “molecular scissors” is slated to make its highly anticipated debut…

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    Expectations For This Clinical Trial Were to Slow Down ALS Progression; Instead it Reversed One Man’s Symptoms

      According to a recent article published in News NWA, in 2017 Mark Bedwell, of Fort Smith, Arkansas had been told that based upon the rapid progression of his amyotrophic…

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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
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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…

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    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…

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    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…

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    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,…

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    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…

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    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…

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